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Severe Combined Immunodeficiency Disease Characterization of the Disease and Results of Transplantation

Mortimer M. Bortin, MD; Alfred A. Rimm, PhD
JAMA. 1977;238(7):591-600. doi:10.1001/jama.1977.03280070031019.
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Pretransplant and posttransplant data for 69 patients with severe combined immunodeficiency disease are presented. Both B and T lymphocyte functions were absent in approximately 80% of the children and markedly depressed in the remainder. Transplantation of marrow from HLA genotypically identical donors provided the highest six-month survival rate (63%); six-month survival rates for patients who received fetal tissue transplants (43%) or marrow from mixed leukocyte culture (MLC) negative donors (38%) were significantly higher (P <.05) than for patients treated with marrow from MLC positive donors (5%). Additional factors appeared to influence survival and the severity of graft-vs-host (GVH) disease. Patients more than 6 months of age had more intense GVH disease than younger patients. Survival rates were lower and GVH disease more intense when boys received transplants from girl donors than the reverse.

(JAMA 238:591-600, 1977)


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