Outcome after unrelated donor bone marrow (BM) transplantation for severe aplastic anemia (SAA) has improved, with survival rates now approximately 75%. Increasing use of peripheral blood stem and progenitor cells (PBPCs) instead of BM as a graft source prompted us to compare outcomes of PBPC and BM transplantation for SAA. We studied 296 patients receiving either BM (n ‫؍‬ 225) or PBPC (n ‫؍‬ 71) from unrelated donors matched at human leukocyte antigen-A, -B, -C, -DRB1. Hematopoietic recovery was similar after PBPC and BM transplantation. Grade 2 to 4 acute graft-versushost disease risks were higher after transplantation of PBPC compared with BM (hazard ratio ‫؍‬ 1.68, P ‫؍‬ .02; 48% vs 31%). Chronic graft-versus-host disease risks were not significantly different after adjusting for age at transplantation (hazard ratio ‫؍‬ 1.39, P ‫؍‬ .14). Mortality risks, independent of age, were higher after PBPC compared with BM transplantation (hazard ratio ‫؍‬ 1.62, P ‫؍‬ .04; 76% vs 61%). These data indicate that BM is the preferred graft source for unrelated donor transplantation in SAA. (Blood. 2011; 118(9):2618-2621)

• Hematopoietic cell transplantation results in long-term survival.• Primary graft failure is very high and the predominant cause of death.We report the international experience in outcomes after related and unrelated hematopoietic transplantation for infantile osteopetrosis in 193 patients. Thirty-four percent of transplants used grafts from HLA-matched siblings, 13% from HLA-mismatched relatives, 12% from HLA-matched, and 41% from HLA-mismatched unrelated donors. The median age at transplantation was 12 months. Busulfan and cyclophosphamide was the most common conditioning regimen. Long-term survival was higher after HLA-matched sibling compared to alternative donor transplantation. There were no differences in survival after HLAmismatched related, HLA-matched unrelated, or mismatched unrelated donor transplantation. The 5-and 10-year probabilities of survival were 62% and 62% after HLA-matched sibling and 42% and 39% after alternative donor transplantation (P 5 .01 and P 5 .002, respectively). Graft failure was the most common cause of death, accounting for 50% of deaths after HLA-matched sibling and 43% of deaths after alternative donor transplantation. The day-28 incidence of neutrophil recovery was 66% after HLA-matched sibling and 61% after alternative donor transplantation (P 5 .49). The median age of surviving patients is 7 years. Of evaluable surviving patients, 70% are visually impaired; 10% have impaired hearing and gross motor delay. Nevertheless, 65% reported performance scores of 90 or 100, and in 17%, a score of 80 at last contact. Most survivors >5 years are attending mainstream or specialized schools. Rates of veno-occlusive disease and interstitial pneumonitis were high at 20%. Though allogeneic transplantation results in long-term survival with acceptable social function, strategies to lower graft failure and hepatic and pulmonary toxicity are urgently needed. (Blood. 2015;126(2):270-276)

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Key Points Autologous hematopoietic cell transplantation is safe and effective in patients with HIV-related lymphoma who meet standard transplant criteria. Patients with HIV-related lymphomas should not be precluded from participating in AHCT clinical trials.

HRQOL for bipolar youth improves following pharmacological treatments. However, distinct effects of specific treatments may exist, and impairment in several domains persists.

AA who developed MDS or AML following IST reported a 5-year event-free survival of 41%. 13 However, there no large-scale study of transplantation for post-AA MDS has been made, and there are no data comparing results to patients with de novo MDS. Thus, in the present study, we analyzed transplant outcomes of post-AA MDS patients, carried out a matched-pair analysis including patients with de novo MDS, and determined prognostic factors for transplant outcome. Methods Study populationIn the databases of the Center for International Bone Marrow Transplantation Research (CIBMTR) and the Fred Hutchinson Cancer Research Center, we identified 123 patients transplanted from 1991 through 2011 for MDS developing after treatment of aplastic anemia with IST (post-AA MDS). Cases of marrow failure/AA possibly associated with constitutional/inherited disorders were excluded. The time interval from the diagnosis of AA to the establishment of the diagnosis of MDS was 4-491 months (median 34 months). The MDS categories comprised refractory anemia (RA), refractory anemia with ring sideroblasts (RARS), refractory anemia with excess blasts (RAEB), and refractory anemia with excess blasts in transformation (RAEB-t). The data bases did not allow for categorization according to the WHO classification, nor was it possible in this retrospective analysis to categorize patients according to the recently proposed five cytogenetic risk groups as used in the revised International Prognostic Scoring System (IPSS-R).14 Patients with AML (>30% marrow myeloblasts) and CMML were excluded. We conducted a matched pair analysis, matching cases to control on factors shown to influence transplantation outcomes. Overall, 1473 patients with de novo MDS were reported to the CIBMTR during the study period. Patients with MDS related to cytotoxic therapy, Fanconi anemia and other inherited marrow failure syndromes were excluded from the study. Cases were matched to controls from this cohort for age (< 10 years vs. ≥ 10 years), disease status at transplantation (RA/RARS vs. RAEB/RAEB-t), donor type (HLA-identical sibling vs. other relative vs. matched unrelated donor vs. mismatched unrelated donor), year of transplant (1991-1999 vs. 2000-2011), and conditioning regimen intensity (myeloablative vs. reduced intensity). Cases and controls were matched to their respective age as close as possible: the difference in age of cases and controls was 0-1 year for 56%, 2-5 years for 30%, and 6-10 years for the remaining 14%. For this latter sub-group, the median age for cases was 23 years, compared to 29 years for controls. DefinitionsCytogenetic risk was graded into good, intermediate, and poor, based on the IPSS.14 Time of engraftment was defined as the first of three consecutive days with neutrophil counts that exceeded 0.5 x10 9 /L. Acute and chronic graft-versus-host disease (GvHD) were defined and graded according to standard criteria. 15,16 Overall survival (OS) was defined as survival irrespective of disease status, and relapse-free survival (RFS) as sur...

Reduced intensity conditioning/non-myeloablative conditioning regimens are increasingly used in allogeneic hematopoietic cell transplantation (HCT). Reports have shown CD34+ dose to be important for transplant-outcome using myeloablative conditioning. The role of CD34+ dose of peripheral blood progenitor cells (PBPC) has not been previously analyzed in a large population undergoing reduced intensity conditioning/non-myeloablative HCT. We studied 1,054 patients aged 45–75 years, with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) transplanted between 2002 and 2011. Results of multivariate analysis showed that PBPC from HLA-matched siblings containing <4 × 106 CD34+/kg were associated with higher non-relapse mortality (HR 2.03, p=0.001), overall mortality (HR 1.48, p=0.008), and lower neutrophil (OR 0.76, p=0.03) and platelet (OR 0.76, p=0.03) recovery. PBPC from unrelated donors with CD34+ dose <6 × 106 CD34+/kg were also associated with higher non-relapse (HR 1.38, p=0.02) and overall mortality (HR 1.20, p=0.05). In contrast to reports after myeloablative HCT, CD34+ dose did not affect relapse or graft-versus-host disease with either donor type. An upper cell dose limit was not associated with adverse outcomes. These data suggest that PBPC CD34+ dose >4 × 106 CD34+/kg and >6 × 106 CD34+/kg are optimal for HLA-matched sibling and unrelated donor HCT, respectively.

Survival for children with relapsed T-ALL is poor when treated with chemotherapy alone and outcomes after allogeneic hematopoietic cell transplantation (HCT) is not well described. Two hundred and twenty-nine children with T-ALL in second complete remission (CR2) received a HCT following myeloablative conditioning between 2000–2011 and were reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). Median age was 10 (range, 2–18) years. Donor source was umbilical cord blood (26%), matched sibling bone marrow (38%) or unrelated bone marrow/peripheral blood (36%). Acute GVHD (grade 2–4) and chronic GVHD occurred in 35% (95% CI, 27–45) and 26% (95% CI, 20–33) of patients. Transplant related mortality at day 100 and 3-year relapse rates were 13% (95% CI, 9–18) and 30% (95% CI, 24–37) respectively. Three year overall survival and disease-free survival were 48% (95% CI, 41–55) and 46% (95% CI, 39–52%) respectively. In multivariate analysis, patients with bone marrow relapse, with or without concurrent extramedullary relapse prior to HCT, were most likely to relapse (HR=3.94, p=0.005) as compared to isolated extramedullary disease. In conclusion, HCT for pediatric T-ALL in CR2 demonstrates reasonable and durable outcomes and consideration for HCT is warranted.