Bone marrow Transplantation (BMT) is a life-saving procedure for the cure of many life-threatening disorders of children and adults1. In low- and middle-income countries in Africa, Middle East and South Asia the major indication for BMT are hemoglobinopathies, i.e. sickle cell disease (SCD) and severe thalassemia (ST), followed by aplastic anemia, leukemias and lymphomas. Moreover, BMT has the potential to cure other severe disorder deriving from congenital or acquired abnormalities of blood cell formation or immune function, including HIV infection and is increasingly cost effective for ST and possibly also for SCD. BMT is the only available definitive cure for SCD with reported success rates in excess of 90% in children having a compatible sibling. SCD is the most common non-communicable life-threatening disease of children in most Sub-Saharian African countries and a growing burden of disease. Only in Zambia there are an estimated 50,000 - 60,000 SCD cases based on a 20-30% prevalence of healthy carriers (SCD traits), and it has been estimated that if SCD patients reach only half the African life expectancy norm, there will be 6 million affected children in that continent.
As a result of a visit in Lusaka from January 8-11, 2013, Dr Lawrence Faulkner, BMT expert and medical coordinator of C2C, believes that all major technical and professional BMT start up requirements are in place or could be implemented within 3 to 6 months provided there is the appropriate local commitment and governmental funding. C2C commits to provide free on-site training and on-line ongoing 24/7 assistance with its team of BMT experts.
Vision for this Project
To develop one or more centers of excellence in Zambia providing BMT with same or better outcomes compared to global quality standards but with reduced costs, thus leading to international competitiveness and sustainability. BMT for SCD is a unique opportunity to strengthen tertiary health care as well as higher medical education. A critical component of the success of this project will be to develop a cascade or targeted screening program aiming at community outreach and early identification of SCD patients, i.e. before irreversible disease-related organ damage occurs.
Mission for this Project
To cure, and thus improve quality and quantity of life, to as many children as possible with sickle cell disease and other severe disorders in Zambia.
- Develop 3 pilot screening, counseling and early identification programs in Zambian communities with a known high SCD prevalence.
- Develop a family support program as well as social entrepreneurship activities involving needy families and contributing to sustainability.
- Perform at least 10 BMTs in 2013 and 30 in 2014, and assess critical outcome (cure rates, mortality, morbidity and quality of life).
- Aim from the very beginning at implementing quality assurance programs and international accreditation standards of excellence.
- Involve all available potential governmental and non-governmental stakeholders.
- Implement early identification of affected children in parallel with family support programs.
- Rely on successful experiences and procedures consolidated in comparable settings.
- Implement shared criteria and procedures to identify and assess appropriate BMT candidates.
- Validate BMT-specific essential tests and procedures.
- Develop procedures for the procurement of critical drugs, reagents and other supplies.
- Design and develop one or more start up BMT units in Lusaka sharing common procedures and protocols.
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