22 Dec

In many countries, particularly in South Asia, where the majority of the pediatric population of our planet lives, a significant, and increasing, proportion of healthcare-related expenses is employed for the non-curative supportive care of patients with severe thalassemia. Major advances in effective oral chelators have heralded a new thalassemia era in which patients are expected to have long and productive lives provided access to long-term appropriate care is available. Unfortunately, this is not always the case for most thalassemics, largely due to costs; appropriate life-long supportive therapy is estimated to cost a minimum of 7.000 USD/year, which is higher than the average income in many thalassemia-prone regions. Moreover, drug or transfusion intolerance, blood-borne infections, osteoporosis and pain, pulmonary hypertension and health-related quality of life (HRQoL) remain some open questions. In parallel with improvements in supportive care also hematopoietic stem cell transplantation (blood and marrow transplantation or BMT) results have substantially improved over the last decade so that a low-risk patients with a compatible sibling is expected to enjoy over 80% cure probability with improved HRQoL. As opposed to supportive care, however, BMT costs have decreased to the point that it is possible to perform a bone marrow transplant for 15.000 USD, i.e. what is needed for 1-2 years of non-curative supportive care, thus making BMT highly cost-effective. Both the transplant community and international accreditation bodies are becoming increasingly aware that expensive and complex hospital infection control standards may not be required. The new thalassemia era has also provided the ability to induce negative iron balance, reverse some of the organ damage associated with chronic iron overload, downstage high-risk patients and further decrease transplant-related morbidity and mortality. Finally, it is become increasingly feasible to use partially matched related donors, e.g. mother of father, which are available for the great majority of patients. This latter option might be safer, more accessible, less expensive and as widely applicable as upcoming gene therapy.

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