The allogeneic hematopoietic stem cell transplant represents the true curative treatment for several types of primary immunodeficiency or primary immune deficiency disorders. This therapeutic approach allows for the reconstitution of the immune system and helps control infections through the introduction of memory T cells.
We conducted a retrospective study, reporting on 13 cases of hematopoietic stem cell transplants performed in various bone marrow transplant units for children with primary immune deficiencies, followed up in the primary immunodeficiency unit of the Pediatrics 1 department at the Children’s Hospital of Casablanca over a period from 2010 to 2024. The aim of our study is to explore the indications, methods and procedures, results, and complications in our series and then compare them with the data and recent series in international literature.
The indication for hematopoietic stem cell transplant was severe combined immunodeficiency in 10 cases, HLA class II deficiency in 2 cases, and combined primary immunodeficiency in 1 case. The average age of our patients at the time of transplantation was 32 months. Add the average transplant delay. In 8 cases, the donor was a fully matched sibling, and in 5 cases, a haploidentical parent. The graft source was marrow in all cases. Eight patients received conditioning, compared to 5 who did not. Graft-versus-host disease prophylaxis was administered in 9 patients in our series, using ciclosporin in all cases, combined with mycophenolate mofetil in 3 cases or with methotrexate in another case. Only three patients developed acute graft-versus-host disease. The outcome was favorable, with stable and satisfactory immune reconstitution in 10 patients. Two deaths were reported after a median delay of 27 days post-transplant, in the context of sepsis. Finally, one patient experienced graft loss 10 years after the transplant, and a second transplant is being considered.
Overall, while our series is limited, the results obtained are very promising. Comparison with results from other series shows many similarities and alignments in terms of outcomes, although efforts are still needed to improve transplant timing, treatment accessibility, as well as follow-up methods, especially chimerism. The therapeutic achievement remains the successful execution of haploidentical transplants, which offer a potential donor for each patient.
