Setting up and sustaining blood and marrow transplant services for children in middle-income economies: an experience-driven position paper on behalf of the EBMT PDWP

Abstract

Severe blood disorders and cancer are the leading cause of death and disability from noncommunicable diseases in the global pediatric population and a major financial burden. The most frequent of these conditions, namely sickle cell disease and severe thalassemia, are highly curable by blood or bone marrow transplantation (BMT) which can restore a normal health-related quality of life and be cost-effective. This position paper summarizes critical issues in extending global access to BMT based on ground experience in the start-up of several BMT units in middle-income countries (MICs) across South-East Asia and the Middle East where close to 700 allogeneic BMTs have been performed over a 10-year period. Basic requirements in terms of support systems, equipment, and consumables are summarized keeping in mind WHO’s model essential lists and recommendations. BMT unit setup and maintenance costs are summarized as well as those per transplant. Low-risk BMT is feasible and safe in MICs with outcomes comparable to high-income countries but at a fraction of the cost. This report might be of assistance to health care institutions in MICs interested in developing hematopoietic stem cell transplantation services and strengthening context appropriate tertiary care and higher medical education.

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

“…However, for a variety of reasons only a small minority of patients receive BMT ( Bolaños-Meade and Brodsky, 2014 ): including in the US, where the average cost of this procedure is in the range of $200,000–400,000. A survey of three sites in LMICs yields instead a cost of less than $15,000 ( Faulkner et al, 2021 ). Based on this last figure, if a child were diagnosed with SCD at the age of 2, and if she/he were to take HU regularly (see below), by the age of 50 the expenditure on HU would be roughly the same as if BMT had been carried out at the time of diagnosis.…”

“…HU ameliorates SCD through at least two different mechanisms (see Figure 1 ). On one hand, by inhibiting cell division in the later stages of erythropoiesis, it favours the synthesis of foetal haemoglobin (Hb F): insertion of a Hb F molecule hinders polymerization of deoxy-Hb S, thus decreasing sickling ( Faulkner et al, 2021 ). On the other hand, it decreases the neutrophil count, thus reducing the detrimental effects of the chronic inflammatory state that tends to be permanent in SCD patients ( Harif et al, 2020 ).…”

Precursor B‐cell acute lymphoblastic leukaemia—a global view

“…The definition of the upper paediatric age limit in oncology is quite variable, being generally lower in non‐HIC compared to HIC, probably due to the different age distribution. While in Ireland patients up to the age of 16 years are treated in a paediatric institution, in the other countries this 15‐year‐old girl would be treated in an adult ward (possibly not in India), where, however, lately there has been a progressive trend to treat AYA patients with paediatric‐oriented protocols 71 . This aspect thus should not substantially change her treatment.…”

Transplantation and Cellular Therapy

“…With this foundation, HCT programs can evolve as they gain experience and grow and can add on personnel and infrastructure as they care for increasingly complex patients and perform more sophisticated transplantation procedures. Other guidelines are also available that can be used as a platform to define the required elements as a new HCT program is considered [17].…”