{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T05:26:13Z","timestamp":1761974773938},"reference-count":21,"publisher":"BMJ","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Arch Dis Child"],"published-print":{"date-parts":[[2001,6,1]]},"abstract":"<jats:sec><jats:title>BACKGROUND<\/jats:title><jats:p>CD40 ligand (CD40L) deficiency is a rare X linked immunodeficiency disorder leading to recurrent bacterial infection, with cryptosporidial enteritis and subsequent hepatic cirrhosis. Bone marrow transplantation offers the only cure.<\/jats:p><\/jats:sec><jats:sec><jats:title>OBJECTIVE<\/jats:title><jats:p>To analyse retrospectively the outcome of bone marrow transplantation for this condition in one centre.<\/jats:p><\/jats:sec><jats:sec><jats:title>DESIGN<\/jats:title><jats:p>A retrospective case note analysis was performed, identifying all patients with CD40L deficiency who had undergone bone marrow transplantation between May 1988 and December 2000. Details of pre-existing infection, pretransplantation immunological and infective data, transplant procedure (particularly donor type and HLA match), conditioning regimen, and marrow manipulation were analysed. Post-transplantation data including infective episodes, engraftment details, immune function, complications, and outcome were recorded.<\/jats:p><\/jats:sec><jats:sec><jats:title>RESULTS<\/jats:title><jats:p>Eight boys (age 1\u201314 years, median 5.75) had transplants. Six received T cell depleted unrelated donor marrow. Four survive and have normal immune function. Six had previous <jats:italic>Pneumocystis carinii<\/jats:italic> pneumonia and three had histological liver damage. Survival was associated with younger age at transplantation and normal liver histology.<\/jats:p><\/jats:sec><jats:sec><jats:title>CONCLUSIONS<\/jats:title><jats:p>Bone marrow transplantation can be curative in CD40L deficiency. Better outcome is associated with younger age at transplantation and normal liver histology.<\/jats:p><\/jats:sec>","DOI":"10.1136\/adc.84.6.508","type":"journal-article","created":{"date-parts":[[2002,7,27]],"date-time":"2002-07-27T10:32:32Z","timestamp":1027765952000},"page":"508-511","source":"Crossref","is-referenced-by-count":41,"title":["Bone marrow transplantation for CD40 ligand deficiency: a single centre experience"],"prefix":"10.1136","volume":"84","author":[{"given":"K","family":"Khawaja","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A R","family":"Gennery","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T J","family":"Flood","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M","family":"Abinun","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A 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