Single-centre experience of allogeneic haemopoietic stem cell transplant in paediatric patients in Cape Town, South Africa

Abstract

Corresponding author: A van Eyssen (ann.vaneyssen@uct.ac.za) Background. Allogeneic haemopoietic stem cell transplant (Allo-HSCT) is a specialised and costly intervention, associated with significant morbidity and mortality. It is used to treat a broad range of paediatric conditions. South Africa (SA) is an upper middle-income country with limitations on healthcare spending. The role of paediatric Allo-HSCT in this setting is reviewed. Objectives. To review paediatric patients who underwent Allo-HSCT at the Groote Schuur Hospital/University of Cape Town Private Academic Hospital transplant unit in Cape Town, South Africa, and received post-transplant care at Red Cross War Memorial Children's Hospital, over the period January 2006 -December 2014 in respect of indications for the transplant, donor sources, conditioning regimens, treatment-related morbidity and overall survival (OS). Methods. A retrospective analysis of patient records was performed and a database was created in Microsoft Access. Descriptive analyses of relevant demographic, clinical and laboratory data were performed. Summary statistics of demographic and clinical parameters were derived with Excel. OS was calculated from the date of transplant to the date of an event (death) or last follow-up using the Kaplan-Meier method in Statistica. Results. A total of 48 children received Allo-HSCT: 24 for haematological malignancies, 20 for non-oncological haematological conditions, 3 for immune disorders and 1 for adrenoleukodystrophy. There were 28 boys (median age 7.5 years) and 20 girls (8.5 years). There were 31 sibling matched peripheral-blood stem cell (PBSC) transplants and 1 maternal haploidentical PBSC transplant. Stem cells were mobilised from bone marrow into peripheral blood by administering granulocyte-colony stimulating factor to donors. PBSCs were harvested by apheresis. Eight patients received 10/10 HLA-matched grafts from unrelated donors. Six were PBSC grafts and 2 were bone marrow grafts. Three of the unrelated PBSC grafts were from SA donors. Eight transplants used umbilical cord blood from international registries. OS for patients with non-oncological disorders was 91.3% (median follow-up 3.9 years), while that for oncology patients was 56.8% (1.9 years). Two of the survivors developed chronic graft-versus-host disease. Conclusions. OS for non-oncological conditions was excellent, while outcomes for oncological disorders were on par with those in highincome settings. Transplantation offers many patients the opportunity for long-term survival and has been shown to be both feasible and rewarding in a less well-resourced environment servicing an economically diverse population.

Haematopoietic stem cell transplantation in South Africa: Current limitations and future perspectives

“…The overall survival in children treated for non-oncological diseases was 91% (median follow-up of 3.94 years) and 57% (median follow-up of 1.86 years) for children treated for haematological malignancies. These observations were comparable to outcomes reported in highincome counterpart facilities, [28] and justify allocating the country's limited resources to additional state-funded programmes.…”

“…The high cost involved in accessing international donors is therefore a limitation, owing to the same reason. [28] The past few years have seen an increase in government-funded hospitals providing HSCT, but there is at present only one academic centre with a fully-fledged HSCT programme for allogeneic (including haploidentical) transplantation. Van Eyssen et al [28] assessed the outcomes of 48 state-funded children who received allogeneic HSCTs at the Groote Schuur Hospital/ University of Cape Town Private Academic Hospital transplant unit.…”

“…[28] The past few years have seen an increase in government-funded hospitals providing HSCT, but there is at present only one academic centre with a fully-fledged HSCT programme for allogeneic (including haploidentical) transplantation. Van Eyssen et al [28] assessed the outcomes of 48 state-funded children who received allogeneic HSCTs at the Groote Schuur Hospital/ University of Cape Town Private Academic Hospital transplant unit. The authors aimed to evaluate the clinical outcomes of allogeneic HSCT in order to motivate future allocation of the substantial resources required to sustain such a treatment programme in SA.…”

A Tanzanian Boy with Molecularly Confirmed X-Linked Adrenoleukodystrophy

“…ere are reports on ALD or its clinical suspicion from South Africa, but cases published do not mention genetic confirmation [6][7][8]. e c.1534G>A (p.(Gly512Ser)) mutation causes a complete loss of function with no detection of ADLP in patient cells [10].…”

“…Mutations in ABCD1 have been described in ALD patients from populations around the world [1], but reports from Africa are limited to a few North African countries. In the Republic of South Africa ALD does occur [6][7][8] although genetic con rmation is not available in scienti c literature.…”