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Allogeneic CD34+ -selected peripheral stem cell transplantation from parental donors in children with non-malignant
1Department of Pediatric Hematology-Oncology and Endocrinology, West German Tumor Center, University Hospital, Essen, Germany.
Insights
Allogeneic stem cell transplants using CD34+-selected grafts from mismatched parental donors successfully engrafted in children with fatal non-malignant diseases. Infections remain a significant threat to survival post-transplant.
Area of Science:
- Pediatric Hematology
- Transplantation Immunology
- Gene Therapy
Background:
- Non-malignant hematologic and metabolic diseases can be fatal in children.
- Allogeneic bone marrow transplantation (BMT) offers a potential cure but donor availability is a challenge.
Purpose of the Study:
- To evaluate the efficacy and safety of CD34+-selected allogeneic peripheral stem cell transplantation (SCT) from mismatched parental donors in children with fatal non-malignant diseases.
- To assess engraftment, graft-versus-host disease (GVHD) incidence, and survival rates.
Main Methods:
- Six children with fatal non-malignant diseases received SCT from parental donors with 3-5 HLA mismatches.
- Grafts underwent CD34+ cell selection for GVHD prophylaxis.
- Median doses of 16.7 x 10(6) CD34+ cells/kg and 1.2 x 10(4) CD3+ cells/kg were administered.
Main Results:
- All six patients achieved successful engraftment, with neutrophil recovery by day 11 and platelet recovery by day 13.
- Three patients developed mild, skin-restricted acute GVHD (grade I), responsive to steroids.
- Four patients survived with stable blood counts at 13-26 months; two died from viral infections.
Conclusions:
- CD34+-selected stem cell transplants from mismatched or haploidentical parents are a viable option for children with fatal non-malignant diseases when no other donor is available.
- High CD34+ cell doses and low CD3+ cell numbers facilitate engraftment and minimize acute GVHD.
- Post-transplant infections remain the primary cause of mortality.
Abstract:
Allogeneic peripheral stem cell transplantation in six children with non-malignant hematologic or metabolic diseases which are eventually fatal was carried out with parental donors. Given three to five HLA mismatches, all grafts underwent CD34+ cell selection as graft-versus-host prophylaxis. The patients received median doses of 16.7 x 10(6) CD34+ cells/kg and 1.2 x 10(4) CD3+ cells/kg. All transplants engrafted. Neutrophils >0.5/nl were reached on day 11 (9-19) and platelets >50/nl on day 13 (10-25). Acute GVHD responding to steriods occured in three of six patients; it was restricted to the skin and overall did not exceed grade I. Two patients died of viral infections and four are alive with stable blood counts for 13, 15, 25 and 26 months. For children with non-malignant diseases which will eventually be fatal and which can be cured or ameliorated by allogeneic BMT, CD34+-selected stem cell transplants from mismatched or even haploidentical parents can be used if no other suitable donor is available. With high CD34+ cell doses and low CD3+ cell numbers, engraftment and avoidance of acute GVHD can be expected. Infections after transplantation remain the primary threat to survival.