Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency.

[severe combined immunodeficiency]

Patients with severe combined immunodeficiency disease who have matched sibling donors (MSDs ) can proceed to hematopoietic cell transplantation (HCT ) without conditioning chemotherapy .We sought to determine whether the results of HCT without chemotherapy-based conditioning from matched unrelated donors (URDs ), either from volunteer adults or umbilical cord blood , are comparable with those from MSDs .We performed a multicenter survey of severe combined immunodeficiency transplantation centers in North America , Europe , and Australia to compile retrospective data on patients who have undergone unconditioned HCT from either URDs (n = 37 ) or MSDs (n = 66 ).Most patients undergoing URD HCT (92 %) achieved donor T -cell engraftment compared with 97 % for those with MSDs ; however , estimated 5 -year overall and event-free survival were worse for URD recipients (71 % and 60 %, respectively ) compared with MSD recipients (92 % and 89 %, respectively ; P < .01 for both ). URD recipients who received pre-HCT serotherapy had similar 5 -year overall survival (100 %) to MSD recipients . The incidences of grade II to IV acute and chronic graft-versus-host disease were higher in URD (50 % and 39 %, respectively ) compared with MSD (22 % and 5 %, respectively ) recipients (P < .01 for both ). In the surviving patients there was no difference in T -cell reconstitution at the last follow-up between the URD and MSD recipients ; however , MSD recipients were more likely to achieve B-cell reconstitution (72 % vs 17 %, P < .001 ).Unconditioned URD HCT achieves excellent rates of donor T -cell engraftment similar to that seen in MSD recipients , and reconstitution rates are adequate . However , only a minority will have myeloid and B-cell reconstitution , and attention must be paid to graft-versus-host disease prophylaxis . This approach might be safer in children ineligible for intense regimens to spare the potential complications of chemotherapy .

Diseases
Validation

Diseases presenting "chemotherapy" symptom

classical phenylketonuria

heparin-induced thrombocytopenia

hodgkin lymphoma, classical

liposarcoma

neuralgic amyotrophy

severe combined immunodeficiency

well-differentiated liposarcoma

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