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Haploidentical bone marrow transplants for two patients with reticular dysgenesis
K B De Santes1, S S Lai, M J Cowan
1Department of Pediatrics, University of California, San Francisco 94143-1278, USA.
Bone Marrow Transplantation
|June 1, 1996
Summary
Reticular dysgenesis (RD) treatment with bone marrow transplant (BMT) requires intensive conditioning. T cell-depleted haploidentical grafts combined with 700 cGy TBI achieved donor engraftment in two RD patients.
Area of Science:
- Immunology
- Hematology
- Pediatric Hematology
Background:
- Reticular dysgenesis (RD) is a rare, severe form of combined immunodeficiency.
- Bone marrow transplantation (BMT) is a potential curative therapy for RD.
- Previous BMT attempts in RD patients have shown variable success rates.
Observation:
- Two cases of RD were treated with T cell-depleted haploidentical marrow grafts.
- Initial conditioning regimens failed to achieve engraftment in both patients.
- One patient failed conditioning with ATG; the other failed multiple regimens including cyclophosphamide and busulfan.
Findings:
- Successful donor engraftment was achieved in both patients after conditioning with 700 cGy total body irradiation (TBI).
- TBI was used with or without additional conditioning agents.
- This suggests a need for more intensive conditioning in RD compared to other severe combined immunodeficiency syndromes.
Implications:
- Intensified conditioning protocols may be necessary for successful BMT in RD patients.
- These findings contribute to optimizing BMT strategies for rare primary immunodeficiencies.
- Further research into conditioning regimens for RD is warranted to improve transplant outcomes.