Thalassemia Family Support program
Table of Contents
3)The Bone Marrow Transplant Team
4)Emotional Aspects of a Bone Marrow Transplant
6)Bone Marrow Transplant Process
8)Communicating with the Doctor
10)Helping Family and Friends Participate in Care
11)Important Telephone Numbers
12)Your Child's Recovering Immune System---Special Precautions
13)General Care/ Adjusting to life at home
14)Commonly Asked Questions During the First Month Following Discharge from the Hospital
15)Follow-up/Outpatient Appointments
Undergoing bone marrow transplant could be a terrifying experience for a family to endure. The life and death struggle affects not only the patient, but the entire family. The necessity to embrace those families is vital to their social, emotional, and financial survival.
The Cure2Children Foundation Family Support Program is a volunteer-based program that provides resources and services to patients and families during their time with the hospital. It was founded in 2007 as a partnership between Cure 2Children foundation and the participating hospital, born through the efforts of families who experienced transplants. The program seeks to embrace families and fully address their needs during the arduous transplant process.
A. Mission
The mission of the Cure2Children Foundation Family Support Program (C2CFSP) is to provide expert, compassionate care to children with thalassemia while advancing the understanding, diagnosis, treatment, cure, and prevention of it. C2CFSP also provides training for health professionals; designs programs that promote public health particularly among high-risk and underserved populations.
B. Vision
C2CFSP's vision is to contribute to the promotion of worldwide communication and collaboration amongst professionals from developing regions to offer the best possible treatment and follow-up care for children with thalassemia and other related diseases. Also, C2CFSP's aim is to foster the interchange of knowledge and management standards that may improve and ultimately decrease barriers to the cure of pediatric severe blood disorder.
C. Goal
More than one in four adults provides care to a family member or friend. However, being a caregiver often presents challenges. The caregiver will need to learn about the disease and its treatment and how to handle medical problems and symptoms. At the same time, they may have to fit in time for doctors' visits and arrange for ways to get the patient to the center for treatment. A partnership, thus, must be made between the team of health professionals, volunteers, and family to make decisions together; share information openly; understand each others point of view; and keep the best interest of the patient as the common goal. Family support program volunteers will use play experience, information about events and procedures, and establish therapeutic relationships with the children and families to support their involvement in the child's care.
The Goal of the C2CFSP is to assist the medical team and patient in removing barriers to care and to help the patient move from one level of care to the next with the least amount of stress. Identification of these barriers and stressors will, thus, provide patient and family with better coping skills and/or compliance with treatment.
A. Definition
The thalassemias are a group of genetic (inherited) blood disorders that share in common one feature, the defective production of hemoglobin, the protein that enables red blood cells to carry oxygen. There are many different disorders with defective hemoglobin synthesis and, hence, many types of thalassemia. The genetic defect underlying thalassemia major, also known as Cooley's disease, results in an imbalance between hemoglobin chains and accumulation of alpha globins chain, leading to intramedullary destruction of erythroid precursors. Patients with thalassemia major present as a progressive and severe form of hemolytic anemia generally during the first year of life.
The most frequent type of thalassemia is beta thalassemia. It involves decreased production of normal adult hemoglobin (Hb A), the predominant type of hemoglobin from soon after birth until death. (All hemoglobin consists of two parts: heme and globin). The globin part of Hb A has 4 protein sections called polypeptide chains. Two of these chains are identical and are designated the alpha chains. The other two chains are also identical to one another but differ from the alpha chains and are termed the beta chains. In persons with beta thalassemia, there is reduced or absent production of beta globin chains.
What is the difference between thalassemia minor and major?
There are two forms of beta thalassemia. They are thalassemia minor and thalassemia major (which is also called Cooley's anemia).
Thalassemia minor: The individual with thalassemia minor has only one copy of the beta thalassemia gene (together with one perfectly normal beta-chain gene). The person is said to be heterozygous for beta thalassemia. Persons with thalassemia minor have (at most) mild anemia (with slight lowering of the hemoglobin level in the blood). This situation can very closely resemble that with mild iron-deficiency anemia. However, persons with thalassemia minor have a normal blood iron level (unless they have are iron deficient for other reasons). No treatment is necessary for thalassemia minor. In particular, iron is neither necessary nor advised.
Thalassemia major (Cooley's anemia): The child born with thalassemia major has two genes for beta thalassemia and no normal beta-chain gene. The child is homozygous for beta thalassemia. This causes a striking deficiency in beta chain production and in the production of Hb A. Thalassemia major is, therefore, a serious disease. The clinical picture associated with thalassemia major was first described in 1925 by the American pediatrician Thomas Cooley. Hence, the name Cooley's anemia in his honor. At birth the baby with thalassemia major seems entirely normal. This is because the predominant hemoglobin at birth is still fetal hemoglobin (Hb F). Hb F has two alpha chains (like Hb A) and two gamma chains (unlike Hb A). It has no beta chains so the baby is protected at birth from the effects of thalassemia major. Anemia begins to develop within the first months after birth. It becomes progressively more and more severe. The infant fails to thrive (to grow normally) and often has problems feeding (due to easy fatigue from lack of oxygen, with the profound anemia), bouts of fever (due to infections to which the severe anemia predisposes the child) and diarrhea and other intestinal problems. Due to anemia, erythropoietin is stimulated causing increased intramedullary and extramedullary erythropoiesis resulting in bone expansion (head bossing) and splenomegaly (these are usually presenting features at the time of diagnosis).
How frequent is thalassemia?
Only in the Mediterranean basin the prevalence of thalassemia is estimated to be in the range of 250.000 and 1 million worldwide. In many countries, due to lack of awareness and/or access to prenatal diagnosis, many new cases of thalassemia are born yearly. It is estimated that in India and Pakistan there are around 50.000 new cases every year.
B. Diagnosis
The clinical diagnosis is relatively simple: a severe chronic anemia manifesting itself between 6 months and 2 years of age in which no beta hemoglobin can be detected on hemoglobin electrophoresis. Both parents should be carriers, i.e. have small red cells (mean corpuscular volume or MCV generally less than 70 fL), many target cells on smear and generally greater than 2.5% Hemoglobin A2 on hemoglobin electrophoresis. In rare instances one can be a carrier with close to normal hemoglobin and red cell size.
Thalassemia minor-Mild anaemia, RBC count will be normal or slightly increased, MCV < 70fl, target cells on blood picture and Hb Electropheresis shows slightly increased HgA2, decreased HgA and slightly increased HgF.
Thalassemia major- Severe anaemia,MCV, MCH, MCHC all are decreased, abnormal red cell morphology and many target cells, Hb Electrophoresis shows decreased HbA and increased HgA2 and HgF.
3. The Bone Marrow Transplant Team
The Bone Marrow Transplant Program has a team of doctors, nurses, other professionals and volunteers, who all work together to provide the best care possible for patients and their families.
A. Attending Physicians
The attending physicians are full-time members of the pediatric faculty with special training and a special interest in bone marrow transplantation. There are hematologists, or blood specialists, as well as immunologists, or experts on the immune system. There are a minimum of two physicians who share overall responsibility for the patients in the Bone Marrow Transplant Unit.
B. Residents and Fellows
Residents are doctors who are in the Pediatric Residency Program and are training to become pediatricians. The residents work with the attending physicians to care for the transplant patients. Fellows have completed a pediatric residency program and are receiving advanced training to become specialists in immunology, hematology or oncology.
C. Clinical Nurse Specialist/Nurse Coordinator
The nurses in the Bone Marrow Transplant Unit are trained to care for the special needs of bone marrow transplant patients. The clinical nurse specialist and nurse coordinator are involved in many aspects of your child's preparation for transplant and treatment in the hospital. This includes:
- Before transplant evaluation and teaching
- Hospitalization and care after transplant
- Outpatient follow-up
The clinical nurse specialist and nurse coordinator work closely with the nursing staff and transplant doctors.
D. Support Staff (see also Manual for Implementation)
Volunteer Coordinator -- The average hospital stay for a bone marrow transplant patient is two months. The VC helps families develop strategies to minimize the disruption caused by this extended hospital stay. This includes providing counseling for all family members about coping with the stresses of bone marrow transplantation. The VC also can provide information regarding lodging, transportation and other community resources that may be helpful.
Volunteers -- Volunteers help children of all ages cope with hospitalization. They provide activities and projects during the hospital stay and work to normal life in the hospital as much as possible for each child. They offer emotional support and encourage patients to express their feelings through play. A volunteer will meet with the donor prior to hospitalization in order to help prepare him or her for the hospital stay and the marrow harvest.
Pharmacist -- Pharmacists assist the health care team in administering and monitoring the effects of the various drugs that are used during and after hospitalization. They also provide teaching and follow-up consultation regarding the patients discharge.
Teachers -- The teacher coordinates a study program during the child's hospital stay and regularly comes to the transplant room to work in various areas of study. After the child is discharged from the hospital, we recommend home tutoring for at least the first six months after the transplant or until the child can return to school.
Laboratory Personnel -- Research associates process and prepare bone marrow stem cells for transplantation. There also are Postdoctoral fellows and research faculty who focus on specific research projects that ultimately will advance the field of knowledge about transplantation.
Other Support Staff -- There are many other professionals who are members of the health care team and who will be involved as needed. They include lab technicians, psychiatrists and physical therapists.
4. Emotional Aspects of a Bone Marrow Transplant
Everyone brings his or her own unique experience and outlook to the bone marrow transplant process. Even though it will be different for everyone who goes through it, some common feelings and concerns seem to be shared by many families. Patients and families can expect that their emotions will seem to flip-flop to opposite extremes. They may feel excited and hopeful to be starting the transplant procedure and, at the same time, feel anxiety about what is ahead. The families may expect to feel tired and frustrated, which can leave them short-tempered at times. Family members often wish they could be at the hospital, at home and at work all at once. Arguments may arise over what other family members are doing, or not doing. Family members may feel guilty or blame each other. Love, anger, vulnerability and faith may all be felt together. Separation from part of their family can be hard. While at some times it may be easy, at other times it may be extremely hard for the child to deal with staying in the isolation room. Complying with the treatment regimen can be difficult and privacy is difficult to maintain since medical care depends on knowing intimate details of everything that happens. We will try to respect the family and child's privacy as much as possible.
5. Planning for the Patients' Stay
A. Housing
Accommodation- accommodation near to transplant center may be provided. This is not available for the patients who are residents of the same city. This is decided on a case to case basis.
B. Parking
Parking will be free for the patient's family ? Hours of operation? Location?
C. Transportation
Pick up and drop off from accommodation can be arranged only for very deserving families.
D. Other Family Members
It is important for the child to be supported by other family members. Other siblings or relatives are welcome to visit during ____________ hours on ____________ days.
E. Packing
When preparing for the child's stay in the hospital. Families should bring a few things that will put the child at more ease such as a favorite stuffed animal, photo, and blanket.
F. Monthly stipend
Only for deserving families coming from other cities and approved on a case by case basis.
6. The Bone Marrow Transplant Process
Bone Marrow Transplantation Treatment
Thalassemia major results from a genetic defect for which the only cure is to replace the abnormal red cell progenitor cells with its normal counterpart. The carrier of the normal gene is the hematopoietic stem cell of a normal individual or a thalassemia carrier (heterozygous). Bone marrow transplantation has been used with success for many years, and implies the intravenous administration of adequate numbers of hematopoietic progenitor cells. Prior to the infusion, the patient is treated with drugs that will both eliminate the abnormal gene containing red cell precursors and prevent rejection of the normal stem cells of the donor. This procedure is called allogeneic transplantation as opposed to autologous transplantation in which the patient's own stem cells are used to repopulate the bone marrow; this latter procedure is not used for genetic defects but for some high-risk malignancies. For allogeneic transplantation, the first-choice donor is generally a family member compatible at the major histocompatibility loci, i.e. the HLA system which is on chromosome 6 and is inherited following Mendelian laws. Thus each sibling has a 25% chance of being HLA-compatible, and the likelihood of having a suitable related donor depends on family size. Even though alternative sources of transplantable stem cells to the bone marrow, such as cord blood or "mobilized" peripheral blood progenitor cells, have been used, marrow still remains the preferred source of stem cells in the HLA-matched transplantation. In fact, cord blood may be associated with prolonged hematological recovery and increased rejection rates, and peripheral blood stem cell transplants with increased risk of chronic graft versus host disease. Bone marrow is generally collected under general or spinal anesthesia by multiple aspirates of the iliac bones.
The bone marrow transplant (BMT) process can, thus, be broken up into six phases. Each of these phases is explained in more detail on the next pages:
Bone Marrow Donors
Stem cells are a type of cell that can grow into other types of cells, including red blood cells, white blood cells, and platelets. They are generated in the bone marrow and found in the bone-marrow even though they can also be found in peripheral blood and umbilical cord blood. Donated stem cells are used to help a recipient produce components of his or her blood compromised by disease.
Auto Donation of blood: Because a significant amount of blood is taken with the marrow, the adult donor may be asked to donate one to two units of his or her own blood, called auto donation, approximately one to two weeks before the harvest. The nurse coordinator or the clinical nurse specialist will make these arrangements. Following the marrow harvest, the donor's blood is infused back into the donor. The goal of this approach is to minimize the need for a blood transfusion from an outside source after the harvest.
Bone-Marrow Donor: Bone-marrow donation involves a minor surgical procedure, which is performed under local or general anesthesia. Four to eight pricks are made in the skin over the pelvic area and used to perform several marrow aspirations with a surgical needle from the pelvic bones. The process takes 45-90 minutes. The patient may initially have soreness at the donation sites, but a full recovery generally occurs within a few days. The donor's system will completely replace the extracted marrow within a few weeks, since marrow continuously regenerates itself.
A. The Transplant Decision -- includes the initial consultation visit, pre-transplant work-up and evaluation and the informed consent conference.
I. Initial Consultation
During the initial consultation visit to the Bone Marrow Transplant Clinic, the child will meet with an attending physician, nurse specialist, volunteer coordinator. The child's history will be reviewed and potential donors discussed. Additional blood studies may be obtained in order to complete the tissue typing. Basic issues of the bone marrow transplant will be reviewed with patients and their families. Following the initial consultation, a decision is made by both parties as to the feasibility and desirability of going ahead with the transplant.
II. Pre-Transplant Work-Up and Evaluation
After the decision has been made to move forward, the next step is for the patient to have a work-up and evaluation. The purpose of the pre-transplant work-up is to thoroughly evaluate the child's medical and psychological status with respect to his or her ability to undergo a transplant. Many potential bone marrow transplant patients have already had significant problems from their primary diseases. These problems as well as their treatment, like chemotherapy, can damage vital organs including the heart, lungs, kidney, liver and brain. Generally, the patient undergoes a thorough medical evaluation two to three weeks prior to admission. This evaluation includes the following tests:
- Echocardiogram
- Developmental Testing
- Dental Evaluation
- Blood Tests -- Blood tests will be performed to evaluate liver and kidney function and to check for certain germs that can cause infections during a bone marrow transplant, such as HIV, CMV (cytomegalovirus), malaria, typhoid fever, tubercolosis, and the hepatitis viruses.
All of these evaluations will be done on an outpatient basis. While the pre-transplant work-up and evaluation is being completed, a tentative schedule is established for admission for transplant. This will vary depending on the child's need for additional treatment prior to transplant and bed availability.
III. Informed Consent Conference
Following the pre-transplant evaluation the patient and their family will have an informed consent conference. They will meet with a bone marrow transplant doctor, nurse, and other members of the health care team to review the results of the pre-transplant evaluation and the treatment plan, and discuss the benefits and risks of a transplant. It is important for the family to take as much time as they need to understand every aspect of the transplant and to have all questions fully answered. The conference takes about an hour and a half, and it is essential that both parents or legal guardians be present. Depending on age and other factors, the recipient and the bone marrow donor also may be present or may have a separate meeting with the BMT team. Prior to admission for the transplant, parents or legal guardians must sign the consent forms. The consent forms are documents that review the essential purposes and procedures of the bone marrow transplant, as well as the risks and benefits. The consent form is signed by the patient and donor, depending on their ages, and their parents as well as the transplant doctor. Signing the consent form indicates that everyone understands as much as possible about what is involved in the bone marrow transplant process and agrees that it is the best treatment available.
B. Before Admission
I. Welcome Bags - Upon admission to the unit, the Family Support Program team will distribute a welcome bag to families. This bag includes phone cards, a brightly colored pillowcase, a colorful laundry bag, a quilt, and several other small items. This is the Family Support Program's way of saying welcome.
II. DVD Screening- The child and family will have the opportunity to watch a brief DVD geared for children which explains the BMT. This helps to ease the fears and worry of the child.
III. Prior to entering the Bone Marrow Transplant Unit, the patient will require the insertion of a central venous catheter which is a special types of intravenous line that remains in place throughout the entire transplant process and help avoid the pain and discomfort of multiple IVs and blood draws. Through this catheter, chemotherapy, medications, blood products and nutritional support will be administered. These special IVs also are used for blood drawing. The catheter is inserted in the operating room. While under general anesthesia, the catheters are inserted into major blood vessels in the neck and exit the skin on the front of the chest or abdomen. Parents and children, depending on their age, will be taught how to care for the catheters and change the dressings. The catheters typically remain in place for three to 12 months after the transplant.
IV.Bone Marrow Harvest: Bone marrow will be obtained from the donor asleep under general anesthesia, multiple marrow aspirations are obtained from the pelvic bones along the lower back.
C. Admission to the BMT Unit - five to 10 days before the transplant, when the patient undergoes a conditioning regimen of high doses of chemotherapy and sometimes radiation therapy.
Transfusion facility in day care- As the child with thalassemia needs transfusions during the pretransplant period, blood should be arranged by the hospital without expecting replacement as it will be difficult for the patient's family to arrange for blood donation when they are out of their hometown. For this purpose, monthly blood camps can be arranged by foundation in colleges, universities etc to ensure the sufficient store of blood products required during pre or post transplant period.
I. The patient and family will be given information such as:
a. The nature of the procedure
b. The goal and its benefits
c. The possible risks
d. What will be done to manage pain and discomfort
e. What would happen if the transplant was not done
f. How the child is prepared for the procedure
Upon admission to the Bone Marrow Transplant Unit, a countdown period of usually five to 10 days begins. Day 0 marks the end of the countdown and the day of transplant. During the countdown period, one of the conditioning regimens is administered. This involves treatment with chemotherapy agents that eliminate abnormal bone marrow cells.
II. The BMT Room
Patients stay in a special BMT room. The room is cleaned every day, and everyone who enters the room must follow specific procedures to minimize complications associated with the transplant process. Visitors to the BMT Unit must follow certain rules that protect the transplant recipient and help the program run smoothly.
All visitors -- including doctors, nurses, other members of the health care team and family members -- must wash their hands before entering and leaving the room. In addition, there is a clean outer room, called an anteroom that separates the patient's room from the hallway. This isolation policy is intended to minimize the risk of developing an infection during the transplant process. Transplant patients must stay in their room, except for when they need x-rays or other imaging, or procedures that can only be done elsewhere, until the new marrow has begun to work (engraftment). The total amount of time in the Transplant Unit varies, but generally it is four to eight weeks.
III. Conditioning Regimen
As mentioned above, for normal stem cells to engraft the hematopoietic and immune systems of the patient marrow has to be removed (ablated). This process is called "conditioning regimen" and generally relies on drugs, such as busulfan and thiotepa which are very toxic to the patient's abnormal bone marrow, and cyclophosphamide, which is a potent immunosuppressive agent and prevents rejection. These drugs are administered in schedules close to the maximum tolerated doses.
After an appropriate wash-out period designed to avoid exposure of donor stem cells to the conditioning regimen, generally 24 to 78 hours, depending on the half-life of the agents employed, the marrow inoculum is infused via a central venous line not much differently than a regular blood transfusion. Hematopoietic stem cells, once in the circulation, will naturally home to their bone marrow and will start to regenerate hematopoietic and immune functions.
The overall goals of the conditioning regimen are to:
- Suppress the immune system so that the patient will not reject the new bone marrow
- Make room in the bone marrow for the donor marrow stem cells to grow
- Destroy any residual abnormal stem cell
D. Day of the Transplant (Day 0) -- when healthy marrow stem cells are delivered into the bloodstream via an intravenous tube.
On Day 0, the day of transplant, the child will receive the bone marrow stem cells collected from the donor. The bone marrow stem cells are stored in a special blood transfusion bag and administered to the recipient in his or her room the same way a blood transfusion is given, through the central line. The infusion typically takes one to two hours to complete. The donor marrow stem cells travel in the bloodstream to the bone marrow space where they grow and mature.
I. Special Treatment of Marrow
If the donor and recipient are HLA matched but have different blood types, the marrow is treated to remove most of the red blood cells and minimize a transfusion reaction. This process takes several hours to complete. The resulting marrow is slightly pink and is administered from a blood transfusion bag.
Before the procedure, donors often are a bit frightened of the unknown and feel better after discussing their thoughts and fears with the health care team. Information about donation is discussed at an age-appropriate level. Children who are donors meet with the child life specialist and are given a chance to use puppets and other forms of play.
II. Bone Marrow Donors
Bone Marrow Harvest
During the bone marrow harvest, which usually occurs on the day of the transplant, a small portion of bone marrow is collected from the donor while under anesthesia in the hospital. Because a significant amount of blood is taken with the marrow, the adult donor may be asked to donate one to two units of his or her own blood, called auto donation, approximately one to two weeks before to the harvest. The nurse coordinator or the clinical nurse specialist will make these arrangements. Following the marrow harvest, the donor's blood is infused back into the donor. The goal of this approach is to minimize the need for a blood transfusion from an outside source after the harvest.
One to two weeks prior to the day of transplant, the donor is examined by a doctor:
- Adult donors are seen by an internist, who performs a screening history and a physical
- Sibling donors are seen by the attending transplant physician
On the day before the transplant, the consent for anesthesia and surgery is signed with the attending transplant physician and the donor is evaluated by the anesthesiologist. Paperwork and additional blood tests are a part of the pre-admission process.
After midnight, the donor cannot eat or drink anything. On the morning of the bone marrow harvest or collection, the donor is admitted to the pre-op area on the _______ floor. There will be doctors, nurses and other patients in this area, including the attending transplant physician who will be performing the bone marrow harvest. The operation usually begins around _______ a.m. In the operating room, the anesthesiologist will administer the anesthesia that will put the donor to sleep. The doctors will then collect a small portion of bone marrow from the upper part of the pelvic bones. This process usually takes 45 to 90 minutes, but may take longer depending on the amount of marrow needed.
The donor will wake up in the recovery room before being taken to his or her hospital room. A large bandage will cover the area from where the bone marrow was taken. Between one to six needle marks will be visible on the donor's skin. The donor will be sore following donation and will have some bruising. The amount of pain varies from donor to donor and generally does not last more than a few days. Medications will be given to help relieve the pain. The nurses will be checking the dressings for drainage at the needle sites and change the dressings as needed. Adult donors are admitted to one of the adult medical units, usually on the _______ floor. Sibling donors are admitted to the _______floor. The adult donor usually leaves the hospital one day after surgery. The sibling donor may be able to leave on the evening of surgery.
E After the Transplant -- when the bone marrow stem cells migrate to the bones and begin to produce healthy red cells, white cells and platelets. It is in this critical stage that complications related to chemotherapy may develop. Within hours of the transplant, the new bone marrow stem cells will find their way to the marrow space in your child's bones where they will grow and start making red cells, white cells and platelets. It will take the new bone marrow about two to four weeks to grow, or engraft, in the bones. During this time mouth sores (mucositis), may develop, it is likely the child will have fevers and will need additional antibiotics.
In addition, multiple transfusions will be needed to support platelet and red blood cell counts. All blood products are tested as carefully and completely as possible by the Blood Bank to minimize transfusion reactions and infection. The need for transfusions will decrease as the new marrow stem cells begin working. Daily complete blood counts (CBC) and platelet counts will help the doctors keep track of how well the new marrow is growing. The absolute neutrophil count (ANC) which is the total number of special white cells or neutrophils that fight bacterial and fungal infections must be greater than 500 for three consecutive days before the attending physician will discontinue strict isolation.
I. Side Effects and Complications
Infection, anemia and bleeding are all potential problems after the transplant. These are expected side effects of the conditioning regimen. Other side effects that can be expected during this post-transplant period include:
- Mouth sores
- Sore throat
- Dry mouth
- Hair loss
- Darkening and dryness of the skin
- Diarrhea
Some or all of these side effects may develop. The lungs, liver and kidneys also may show the cumulative effects of chemotherapy, radiation and other drugs used post-transplant. Complications due to organ damage, such as veno-occlusive disease (VOD) of the liver, may be mild and transient or severe and fatal.
The pre-transplant evaluations as well as careful monitoring while in the BMT Unit are intended to minimize serious complications. However, 5 to 10 percent of all children receiving a BMT will die of a complication such as an infection, bleeding, organ failure, generally within the first three months following the transplant. The likelihood of a fatal complication varies depending upon the underlying condition and age of the patient. Most patients experience some nausea and vomiting during the conditioning regimen, which often continues into the post-transplant period. To control the nausea and vomiting there are anti-nausea medications that can be given through the IV. There are several types of drugs that can be used alone or in combination, including Granisetron (Kytril), Ondansatron (Zofran), Dexamethazone (Decadron), Lorazepam (Ativan), etc... Your child's appetite will probably be very poor during the transplant period. Therefore, nutritional support may be provided using special IV fluids called total parenteral nutrition (TPN). Mouth sores and skin breakdown may be a painful side effect of chemotherapy. To relieve the pain, intravenous pain medications such as Morphine can be administered at intervals or as a continuous infusion.
II. Graft-Versus-Host Disease
Graft-versus-host disease (GvHD) is a complication that may occur during the post transplant period in children who receive marrow stem cells from relatives including histocompatible (HLA) matched siblings, as well as those who receive marrow from unrelated donors. GvHD reactions can occur when cells of the donor's immune system, called T lymphocytes, are infused via a blood or platelet transfusion or a bone marrow transplant into the recipient, who has received conditioning therapy. Blood and platelets are irradiated to prevent GvHD, but the donor graft cannot be treated this way since irradiation also would kill the normal bone marrow stem cells.
A. Types and Manifestations GvHD can take two forms, acute and chronic:
- Acute GvHD usually develops within three months of the transplant and occurs in 10 to 20 percent of HLA-matched sibling transplants in children.
- Chronic GvHD usually develops three to 18 months after the transplant and occurs in approximately 10 percent of HLA matched sibling transplants. Patients who get acute GvHD have a 50 percent chance of developing chronic GvHD. Both types often affect the:
- Eyes, causing conjunctivitis, dryness and irritation, itching
- Skin, causing rash and itching
- Liver, causing jaundice and/or hepatitis
- Stomach and intestinal tract, resulting in loss of appetite, cramping pain, diarrhea and vomiting
- Sometimes other organs of the body, including the lungs and kidneys, also are affected. A biopsy of the affected organ is often needed to confirm the diagnosis. GvHD can be very mild or extremely severe. There is a 10 percent to 20 percent chance of dying from GvHD. When GvHD occurs in recipients of HLA-matched sibling transplants it is usually less severe and more responsive to treatment than when it occurs following a mismatched or unrelated donor transplant.
B. Prevention and Treatment
There are several medications that are used to help reduce the occurrence and severity of GvHD as well as to treat it when it does occur. Two drugs that are used frequently to help minimize the reaction are methotrexate and cyclosporin A. These drugs may be given singly or in combination depending upon the type of transplant and the disease. Like all drugs, these medications may have side effects including kidney and liver injury. Drugs which may be used to treat a reaction once it occurs include steroids, such as prednisone or cyclosporin A. In addition, there are several experimental drugs that may be offered if these standard therapies are ineffective.
F. Leaving the Hospital -- preparing for the return home and learning how to care for the child outside of the hospital.
The patient will be discharged from the hospital when he or she has the following:
- An adequate absolute neutrophil count (ANC)
- No infection or fever
The child will need to have his or her nutritional needs met, either through intravenous fluids (TPN), nasogastric tube feeding and/or eating and drinking without supplementation.
The primary caregivers -- usually the parents -- will need to receive complete discharge teaching by the nursing, dietary and pharmacy team. This includes learning the following:
- How to care for the catheter
- Special isolation precautions
- Medication distribution
- Schedule of follow-up doctor visits
- How to meet the child's diet requirement
- Housekeeping precautions
- Other issues about care at home
The child will not be discharged from the hospital until everyone is comfortable with the care at home.
I. Continued Monitoring for Infection
The risk of developing infections lasts for as long as 6 to 18 months following a bone marrow stem cell transplant. The child will be followed closely by the transplant team along with his or her own doctor. Initially, blood will be drawn one to two times a week to monitor the recovering marrow and nutritional state, and the patient will be seen weekly by his or her doctor. As the child's condition stabilizes, the visits will be extended to monthly for the first six months after transplant, then changed to every three months until two years after transplant, and eventually every six to 12 months thereafter. The exact schedule for each patient varies depending on the type of transplant and the patient's clinical situation. At regular intervals, blood will be drawn to evaluate the function of the new immune system and the degree of engraftment, or the percent of donor cells. The special precautions at home generally are necessary for the first three to six months after transplant. Patients who develop graft-versus-host disease (GvHD) may require special attention for a longer period. Each family will be given a detailed discharge handbook that will help with the teaching and serve as a reference at home.
II. Discharge Bags - Upon discharge from the unit, the Family Support Program distributes a discharge bag to families. This bag includes a going home packet, paper towels, disinfectant wipes, a cooler to transport medicine to the clinic, a set of plastic drawers to store supplies, a tray to help sort and organize medicine, and a garbage can.
A. Medication Assistance
If the patient's family can not afford medications, it will be provided free of charge by the program.
B. Out-of-Pocket expenses
Any out of pocket expenses spent by the patient's family directly related to the treatment, may be re-imbursed by the program on a case by case basis.
8. Communicating with the Doctor
It is important that you and the patient understand the planned treatment and the possible side effects. Some people do not feel comfortable talking with the doctor. They may be afraid of asking a silly question. They may be worried that they won't understand what the doctor is saying. Here are some suggestions to help you:
Tips
- If possible, one person should go with the patient to all the doctors' visits. This person should also be the key contact for giving information about the patient to family members and friends. If questions come up, this person should be the one to call the doctor. The patient must tell the doctor that this person is the one whom the doctor should talk with.
- Before each visit, write down key concerns and a list of questions. Speak with other family members so you also know what they are worried about.
Here are some questions the patient's family may want to ask:
- What is the recommended treatment and why? Are there other treatment options?
- How often will treatment be given and how long will it last?
- What are the side effects of the treatment? How can they be managed?
- What are reasons I should call your office?
- At the beginning of the visit, tell the doctor that you have questions to ask. This helps the doctor plan to make time to answer your questions. Be sure to ask your most important questions first.
- Write down the doctor's answers. If you don't understand something, ask the doctor to explain it or to draw a picture, if you think that will help. Keep your notes in a folder with other medical information you have gathered.
- If there is something you still do not understand when you get home, call the doctor's office. The nurse who works with the doctor may also be able to answer many of your questions.
A. Providing educational materials
Materials will be provided in both Urdu and English. Educational booklets or printed material about pre transplant preparation, transplant procedure and post transplant care and complications are a few examples. Information material about benefits, risks, side effects of the therapy and post transplant complications will also be provided and explained.
B. Providing emotional support/ child life
The ultimate goal of child life services is to make children as comfortable as possible before, during and after hospitalization. Our child life specialists help decrease anxiety surrounding diagnosis, treatment and hospitalization by using medical play and explaining treatment and procedures in language appropriate to the patient's age and development. They also teach other family members the coping skills they may need to care for a child with cancer, and work closely with siblings to help them address their concerns. Since the physical limitations and emotional stress of cancer can disrupt a patient's normal social growth and development, our child life specialists strive to provide children with as many normal life experiences as possible during treatment.
Child life facilitates coping and adjustment of children and their families in three primary service areas:
- Play experience
- Information about events and procedures
- Establish therapeutic relationships with children and families to support their involvement in the child's care
Services provided by child life are:
- Family support services and utilizing family strengths in the care of a child.
- Orientation of a child and family to the healthcare experience.
- Utilize developmentally appropriate methods of preparing the child and family for treatments.
- Provide emotional support.
- Through play, interpret the child's interpretation of procedures and treatments.
- Provide parent education on child development.
Child Life-Medical Play
Medical play aids in the continued growth and development of the child emotionally, intellectually, and socially. It provides safe outlets for children's energy and anxiety. It helps children work out feelings of resentment, fear, anger, and frustration. Playing with familiar things or activities helps to minimize the traumatic effects of illness and hospitalization. It gives children the chance o make choices that increase their sense of autonomy and independence. Since most choices are taken away from the child while in the hospital, play allows them to make choices and control over a situation. It gives the child a sense of achievement as well as helps children avoid boredom and to pass the time through constructive play.
Types of Medical Play
- Role Rehearsal: Child often takes the role of the healthcare professional.
- Medical Play Fantasy: No real medical equipment, pretend play with medical themes.
- Indirect Medical Play: Provides opportunities for familiarity, exploration, and education. More structured than play fantasy.
- Medically Related Art: Use art activities to express understanding of and reaction to medical experience.
10. Helping Family, Friends, and Volunteers Participate in Care
It is often not possible for one person to manage all of the patient's needs. Most likely, the family will need to have others help. Allowing other family members, friends, and even volunteers to take part in the patient's care gives them a chance to feel the rewards of care giving. Family and friends may want to help but may not know how. Or, they may not offer to help in ways that the family expects. The following tips will help the patient's parents organize family and friends who are willing to take part in the patient's care.
Tips
- Identify tasks for which they need help, and note them on a calendar.
- Ask family and friends when they are available and what jobs they feel most comfortable doing. Or, contact each person with a specific request. Be as clear as you can so your expectations are understood.
- As they hear back from each individual, note their responses on the calendar to ensure everything is taken care of. Planning ahead will help prevent a crisis.
Clear guidelines about the precautions which should be observed while dealing with patients having gone through chemotherapy will be provided. Volunteers can be selected from the experienced families of patients who have gone through chemotherapy or from near-by colleges and medical universities. Volunteers can be used for spending time with patients, collecting funds from institutes, blood camping etc.
11. Important Telephone Numbers
Hospital _____________________
24 hour number for on call Physician ____________________
In case of emergency________________________
Volunteer Coordinator _______________________
12. The Patient's Recovering Immune System- Special Precautions
Until the patient's immune function completely recovers, one of the major concerns after bone marrow stem cell transplant is infection. It takes approximately 6-12 months and sometimes longer for the other types of white blood cells (T and B lymphocytes) to increase in number and fight infections. The time frame varies with the type of transplant performed and from individual to individual.
About every three months from the day of transplant, blood will be drawn to check the patient's immune recovery. T lymphocyte recovery occurs before B-cell recovery. The T-cells help fight fungal and viral infections. The B-cells make antibodies, which also help the body fight infections. Until T-cell recovery occurs, the child will have to take several precautions in order to decrease his/her chance of potential infections. This includes the use of several antimicrobial medications such as Septra and Acyclovir.
These precautions help decrease the risk of potentially fatal infections, while maintaining a normal family lifestyle. In spite of all these precautions, infections may develop or exposure may occur which are beyond anyone's control. Our goal is to minimize their occurrence. In general, once the child's T-cells grow back and are functioning properly, he/she can stop isolation precautions, return to work/school, stop taking the preventative medications and resume a normal life.
There are several general precautions taken to decrease the patient's risk of being exposed to potential infections. Here are some general guidelines:
-Avoid Crowds
-Enclosed shopping centers
-Grocery stores
-School
-Movie theaters
-Restaurants
-Parties
-Airplanes
Avoid contact with visitors and friends who are or have recently been ill. Screen all visitors for:
-Runny nose
-Cold sores
-Sore throat
-Diarrhea
-Vomiting
-Fever
-Rashes
-Red or runny eyes (conjunctivitis)
-Chicken pox exposure
-Infants who have had oral polio vaccination (3-4 weeks prior)
Screen all visitors, especially children
If a family member becomes ill, he/she should limit contact with the child until they recover from the illness. It is not necessary for the ill family member to stay away from the home.
If a family member is exposed to or gets chicken pox, TB (tuberculosis), herpes, hepatitis, they must contact the doctor immediately.
Good hand washing is the best way to prevent the spread of infections. Supervise and teach children to wash their hands after using the toilet, after playing with toys or pets and especially before eating.
13. General Care /Adjusting to life at home
About one week before the child is to return home from the hospital, the patient's house will require a thorough "Spring Cleaning." The goal of the cleaning is to minimize the amount of dust, which carries fungus and bacteria that are in the environment. Use common sense. IT IS UNNECESSARY AND IMPOSSIBLE TO CREATE A STERILE ENVIRONMENT.
The family will need to do the following:
- Vacuum all floors, rugs and furniture.
- Wash down all surfaces, including walls, ceilings, windows, appliances, counter tops, floors, etc. with a disinfectant (i.e. Lysol, bleach).
- Dust all surfaces and corners.
- Clean tubs, toilets and sinks with abrasive cleaners and disinfectants.
- Clean or purchase new filters for forced air heaters.
- Wood stoves and Swamp coolers should be avoided.
- Change shower curtain and continue to clean it with disinfectant regularly (every other week). Always clean the tub before the child uses it.
Be sure to keep all cleaning agents out of the reach of young children.
After the child comes home, continue to regularly clean the home at least one to two times per week. Do not allow the child to be present while the parent dusts or vacuums. Have the child in a separate room with the door closed while cleaning.
The child should have his/her own towels, which should be changed regularly (twice a week). Bed linens should be changed once a week. Clothing may be laundered in the usual fashion with other family member's clothing. If a family member's job exposes his/her clothes to an unusual amount of dirt or dust, they must be washed separately.
If the family does not own their own washer and dryer and bring their laundry to a Laundromat, Lysol must be added to each load.
Hand Washing: This is the most important measure against infection. Hands should be washed thoroughly for at least 30 seconds. Using a germicidal soap is recommended.
Food: The desired goal is to promote nutrition while minimizing infection risk. In general, the most preferred foods should be offered. Everything that is well cooked is generally safe. Meals should be prepared after thorough hand-washing and cleaning of cooking tools and surfaces. Freezing should occur within two hours of cooking. Raw food should be avoided and milk should be pasteurized. Fruit should be washed and peeled. It is recommended to drink water from sealed bottles rather than from the tap. Milk and fruit juices should be refrigerated after opening. These recommendations should be followed until on immune suppressive therapy, in general 6 to 8 months after the transplant.
Monitoring for Potential Infections: Make sure the families have a thermometer at home and know how to correctly read and use it. It is important that the parents/volunteers know when the temperature is not normal and whom and when to call.
Mouth Care: Review mouth care routine for home. After discharge from the hospital the child should brush his/her teeth with a soft bristle toothbrush after meals and at bedtime. Notify the doctor if you notice sores, reddened areas, any white patches or bleeding in the child's mouth or on the lips. Every two weeks discard toothbrush and replace with a new one to prevent infection.
School: School should be avoided until the child has been given an ok from the medical staff. Generally, this may take between 6 to 12 months.
14. Commonly Asked Questions During the First Month Following Discharge from the Hospital
Why do my Child's legs hurt?
- When the muscles in your child's legs get tired, they hurt. It takes a while to rebuild muscles and strength after not exercising/walking as usual and being in bed. If this problem does not go away, please call.
When will my child feel hungry?
- Taste buds have changed from the chemotherapy. Some foods you like "don't taste the same." You child's stomach is not used to having food, so it takes time to build back his/her appetite. It may take several months for your child's appetite to return to normal.
When will my child start gaining weight?
- It takes awhile. As you child's appetite returns so will the pounds.
When can my child stop taking these medications?
- Most of the medications decrease the risk of infections (Bactrim, Mycostatin, Acyclovir). Once your child has regained sufficient T-cell function (usually six months post bone marrow stem cell transplant) you will be able to stop these medications.
When can my child's friends visit?
- It is preferred that only a couple of friends, whom you have screened, visit at one time. Healthy visitors are welcome once you return home.
Can my child take a walk or go to the park?
- Yes. Plan it at a time during the day when few children will be at the park. Avoid other children.
Can my child go to the movies?
- Unfortunately no, because you cannot screen people in a move theatre for colds. Try renting a video/DVD and invite a couple of screened friends over for a movie night. Have lots of popcorn.
When will my child's hair grow back?
- It will take 3-6 months for your child to grow his/her hair back.
How clean should my house really be?
- Think of the initial cleaning as a"spring" cleaning. Dust has probably built up during the long hospitalization.
Can we go to others' houses?
- This should be limited to close family homes where a certain amount of cleaning has occurred to reduce the amount of dust. Please discuss further with your physician and clinical nurse specialist.
Can my child eat yogurt?
- Yes, yogurt with active cultures may be eaten provided it is made from pasteurized milk. Use only products manufactured by major companies (i.e. Dannon, Yoplait, Lucerne, Lady Lec, etc.) Frozen yogurt, purchased from a major fast-food-type chain (e.g. Penguin, MacDonald's etc...) may also be eaten.
Can my child be in the same room/house with someone who smokes?
- NO, Avoid secondary smoke since it is not healthy for you or your child. This means that no one can smoke in your house even if your child is not present at the time.
When can my child go back to school/work?
- When they are taken off "isolation". (When T-cells recovery occurs).
Should family members get flu vaccines?
- Yes, all family members should get the flu vaccine except for the patient. (patients will need the flu vaccine when they are ready to be immunized.)
15. Follow-up/Outpatient Appointments
A calendar (which will be given to patients in their welcome bags) will have the next appointments in them.
Additional resources will be given to patients in their welcome and discharge bags.
The British Columbia Children's Hospital
Thalessemia Site
The University of California, San Francisco Children's Hospital
| Attachment | Size |
|---|---|
| Family Support Program Manual for Implementation.pdf | 127.71 KB |
Added Thu, 06/03/2008 - 16:16, last modified 17/06/2008
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