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

Dvorak CC ，Hassan A ，Slatter MA ，Hönig M ，Lankester AC ，Buckley RH ，Pulsipher MA ，Davis JH ，Güngör T ，Gabriel M ，Bleesing JH ，Bunin N ，Sedlacek P ，Connelly JA ，Crawford DF ，Notarangelo LD ，Pai SY ，Hassid J ，Veys P ，Gennery AR ，Cowan MJ ... -  《-》

[A comparative study of unrelated donor and matched-sibling donor allogeneic hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia].

Zhou J ，Fu YW ，Liang LJ ，Wang Q ，Han LJ ，Zu YL ，Zhang Y ，Zhu XH ，Yu FK ，Fang BJ ，Wei XD ，Song YP ... -  《-》

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

Hassan A ，Booth C ，Brightwell A ，Allwood Z ，Veys P ，Rao K ，Hönig M ，Friedrich W ，Gennery A ，Slatter M ，Bredius R ，Finocchi A ，Cancrini C ，Aiuti A ，Porta F ，Lanfranchi A ，Ridella M ，Steward C ，Filipovich A ，Marsh R ，Bordon V ，Al-Muhsen S ，Al-Mousa H ，Alsum Z ，Al-Dhekri H ，Al Ghonaium A ，Speckmann C ，Fischer A ，Mahlaoui N ，Nichols KE ，Grunebaum E ，Al Zahrani D ，Roifman CM ，Boelens J ，Davies EG ，Cavazzana-Calvo M ，Notarangelo L ，Gaspar HB ，Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and European Society for Immunodeficiency ... -  《-》

T-cell-replete haploidentical HSCT with low-dose anti-T-lymphocyte globulin compared with matched sibling HSCT and unrelated HSCT.

We developed an approach of T-cell-replete haploidentical hematopoietic stem cell transplantation (HSCT) with low-dose anti-T-lymphocyte globulin and prospectively compared outcomes of all contemporaneous T-cell-replete HSCT performed at our center using matched sibling donors (MSDs), unrelated donors (URDs), and haploidentical related donors (HRDs). From 2008 to 2013, 90 patients underwent MSD-HSCT, 116 underwent URD-HSCT, and 99 underwent HRD-HSCT. HRDs were associated with higher incidences of grades 2 to 4 (42.4%) and severe acute graft-versus-host disease (17.2%) and nonrelapse mortality (30.5%), compared with MSDs (15.6%, 5.6%, and 4.7%, respectively; P < .05), but were similar to URDs, even fully 10/10 HLA-matched URDs. For high-risk patients, a superior graft-versus-leukemia effect was observed in HRD-HSCT, with 5-year relapse rates of 15.4% in HRD-HSCT, 28.2% in URD-HSCT (P = .07), and 49.9% in MSD-HSCT (P = .002). Furthermore, 5-year disease-free survival rates were not significantly different for patients undergoing transplantation using 3 types of donors, with 63.6%, 58.4%, and 58.3% for MSD, URD, and HRD transplantation, respectively (P = .574). Our data indicate that outcomes after HSCT from suitably matched URDs and HRDs with low-dose anti-T-lymphocyte globulin are similar and that HRD improves outcomes of patients with high-risk leukemia. This trial was registered at www.chictr.org (Chinese Clinical Trial Registry) as #ChiCTR-OCH-12002490.

Luo Y ，Xiao H ，Lai X ，Shi J ，Tan Y ，He J ，Xie W ，Zheng W ，Zhu Y ，Ye X ，Yu X ，Cai Z ，Lin M ，Huang H ... -  《-》

Hematopoietic cell transplantation for children with acute lymphoblastic leukemia in second complete remission: similar outcomes in recipients of unrelated marrow and umbilical cord blood versus marrow from HLA matched sibling donors.

Smith AR ，Baker KS ，Defor TE ，Verneris MR ，Wagner JE ，Macmillan ML ... -  《-》