Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T cells with anti-CD6 mAb.
BACKGROUND
Between 1981 and 1995, 20 children with severe combined immunodeficiency (SCID; median age at transplant, 6.5 [range, 0.5-145] mo, 12 with serious infection) were treated with haploidentical T cell-depleted (anti-CD6 antibody) bone marrow (median number of 5.7 [0.8-18.8] x 10(8) nucleated cells/kg) from mismatched related donors (MMRDs), and 5 children with SCID (median age at transplant, 1.8 [0.5-5.0] mo, 1 with serious infection) were given unmanipulated bone marrow from matched related donors (MRDs). No conditioning or graft-versus-host disease (GvHD) prophylaxis was used.


OBJECTIVE
To assess the outcomes of patients with SCID who received bone marrow from MMRDs or MRDs.


METHODS
We reviewed the medical records of these 25 consecutive patients with SCID (4 with Omenn syndrome).


RESULTS
Of the 20 patients who received bone marrow from MMRDs, 12 engrafted, 10 survived at a median age of 15.2 [10.0-19.1] years, 4 had chronic GvHD (lung, intestine, skin), 5 required intravenous immunoglobulin, and 8 attended school or college. Two of 5 patients who died had chronic GvHD, and 2 developed lymphoproliferative disease. Of the 5 patients who received bone marrow from MRDs, 5 engrafted, 5 survived at a median age of 23.3 [18.5-26] years, 1 had chronic GvHD (lung, skin), 2 required intravenous immunoglobulin, and 4 attended school or college.


CONCLUSIONS
Treatment of critically ill patients with SCID with anti-CD6 antibody T cell-depleted MMRD marrow resulted in an overall 50% long-term survival of patients (83% survival of those engrafted). The principal barriers to long-term survival were delay in diagnosis, life-threatening infection, failure to engraft, and chronic GvHD. Educational goals were achieved in most of the survivors.
Patel
Niraj C
NC
Department of Pediatrics, Section of Allergy and Immunology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX 77039, USA.
Chinen
Javier
J
Rosenblatt
Howard M
HM
Hanson
Imelda C
IC
Brown
Betty S
BS
Paul
Mary E
ME
Abramson
Stuart L
SL
Ritz
Jerome
J
Shearer
William T
WT
D000293:Adolescent; D000328:Adult; D000911:Antibodies, Monoclonal / Q000494:pharmacology*; D015703:Antigens, CD / Q000494:pharmacology*; D000945:Antigens, Differentiation, T-Lymphocyte / Q000494:pharmacology*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006084:Graft Rejection / Q000401:mortality; D006085:Graft Survival / Q000187:drug effects; D006086:Graft vs Host Disease / Q000401:mortality; D006801:Humans; D007223:Infant; D008212:Lymphocyte Depletion; D008232:Lymphoproliferative Disorders / Q000401:mortality; D008297:Male; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000401:mortality* / Q000628:therapy*; D015996:Survival Rate; D013601:T-Lymphocytes; D013997:Time Factors
D000911:Antibodies, Monoclonal; D015703:Antigens, CD; D000945:Antigens, Differentiation, T-Lymphocyte; C062279:CD6 antigen