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. 2009 Dec;155(6):834-840.e1.
doi: 10.1016/j.jpeds.2009.07.049. Epub 2009 Oct 9.

Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis

Mary Dell Railey  1 , Yuliya Lokhnygina, Rebecca H Buckley
Affiliations

Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis

Mary Dell Railey et al. J Pediatr. 2009 Dec.

Abstract

Objective: To determine long-term health benefits of nonablative bone marrow transplantation for severe combined immunodeficiency (SCID), we investigated our cohort of 161 related donor bone marrow-transplanted patients with SCID. Only 16 (10%) had HLA-identical donors.

Study design: All 124 survivors were sent questionnaires about their current clinical statuses. Details from clinic visits were also compiled. One hundred eleven patients (90%) were reached. We compared outcomes of patients transplanted before and after 3.5 months of life and by molecular defect.

Results: The overall survival rate was 77%, but the rate for the 48 infants transplanted in the first 3.5 months of life was 94%, compared with 70% for the 113 transplanted after 3.5 months (P = .002). Twenty-eight (76%) of the 37 deceased patients died of viral infections present at diagnosis. One or more clinical problems were reported to have been present in the past 2 years in 71 (64%) of the survivors, although 95 (86%) were considered healthy by their families.

Conclusions: Most patients with SCID transplanted with related donor marrow without pretransplant chemotherapy have done well in the long term, but those transplanted at <3.5 months of age had a superior survival rate, a lower rate of clinical problems, less need for booster transplants, and better nutritional status.

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Conflict of interest statement

The authors declare no conflicts of interest.

Figures

Figure 1
Figure 1
Kaplan Meier survival curve for all 161 patients with SCID transplanted over a 26 year period. Twenty-two of the patients died in the first year and 13 more between 1 and 5 years post-transplantation, and 28 (75%) of the 37 patients who died succumbed to viral infections present at the time of transplant.
Figure 2
Figure 2
A comparison of clinical features reported as percentages of 41 surviving patients with SCID who were transplanted prior to 3.5 months of age with the same features reported as percentages of 70 surviving patients with SCID who were transplanted after 3.5 months of age and followed long-term for up to 26 years after transplantation.
See this image and copyright information in PMC

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