The Rising Global Burden of Hemoglobinopathies, A Challenge and an Opportunity for Health Care in Pakistan

Abstract

Severe hemoglobinopathies, namely thalassemia major and sickle cell disease, are the most frequent lifethreatening non-communicable disease of children globally: A minimum estimate of 300.000 newborns yearly have some symptomatic globin disorder, these births occur largely in low- and middle-income countries (LMICs) where prevention and management programs are often lacking or insufficient1,2. Hematopoietic stem cell transplantation, also known as blood or marrow transplantation (BMT), is the only established curative modality with success rates over 85% in low risk children with a compatible sibling3–5, moreover, BMT can normalize long-term health-related quality of life (HRQoL)6,7 and be highly cost effective8,9. However, there is a dire shortage of BMT centers in hemoglobinopathy-prone regions10 which often fall in the low- and middle-income country (LMIC) strata, so that many families have to migrate to affluent countries seeking cure for their beloved ones; this not only aggravates misery, psychological and economical burden but perpetuates the hemorrhage of professional and financial resources to high-income countries (HIC).