Search

s / Biol Blood Marrow Transplant 21 (2015) S206eS239 S216 following criteria: (1) ejection fraction (EF) 50% or less (2) fractional shortening (FS) two standard deviations below the age adjusted mean (3) >10% decrease in EF or FS at day 100 from baseline. Results: 92 of 100 consecutive patients completed day 100 screening (8 died). 25% (23 of 92) had LV dysfunction at day 100 (Table). 96%(22 of 23) were asymptomatic at time of screening. Patients with LV dysfunction were older at the time of SCT than those without (p1⁄40.068). Patients receiving PBSC grafts had an increased incidence of LV dysfunction (p1⁄4 0.032), likely related to the diagnosis of those patients (malignancy receiving autologous SCT and Fanconi Anemia). Overall, however, diagnosis was not significantly associated with LV dysfunction. Patients with previous anthracycline exposure had increased LV dysfunction compared to those without (48% vs. 26%). GVHD, engraftment syndrome, and viremias in the first 100 days were not significantly associated with decreased RV function. Death at 1 year was similar in both groups. Discussion: 25% of patients showed signs of cardiac dysfunction at day 100, which was higher than anticipated. Although there were no acute differences in outcome, the long-term complications are unknown. Echocardiography at day 100 identifies SCT cardiac dysfunction, however, more research is needed to understand the overall impact on longterm outcome.

Total number of patients 22 Diagnosis Shwachman-diamond syndrome 8 Aplastic anemia/marrow failure syndrome 7 Dyskeratosis Congenita 3 Seckel syndrome 1 Kostmann’s syndrome 1 Sideroblastic anemia 1 Congenital Macrothrombocytopenia 1 Age in years at HCT 7.27(0.7 -27) Donor source MSD 6 URD 15 UR cord 1 Stem cell source Marrow 20 PBSC 1 Cord 1 Time to neutrophil engraftment (days) 12 (10-48) Mixed chimerism 1 Resolved 1 GVHD Acute GVHD (Gr II-III) 3 Chronic GVHD (skin) 3 Overall outcome Alive 18 Deceased 4 Causes of and post-transplant day of death Liver failure 668 Disseminated fungal infection 264 Coccidoidomycosis 35 Multiorgan failure 154 Abstracts / Biol Blood Marrow Transplant 19 (2013) S233eS256 S252

s / Biol Blood Marrow Transplant 19 (2013) S109eS128 S123 for whom HCT is appropriate, ii) the choice of the different donor sources. A risk-adapted treatment strategy is crucial to improve the outcome of pts with HRHD. Our policy is to offer a haploidentical HCT to adult pts lacking a matched donor in the appropriate time according to clinical indications (www.leukemianet.org, www.ebmt.org). This policy is integrated in ongoing protocols for primary disease. Methods: Here we are reporting the intention-to-treat (ITT) analysis of HCT in all consecutive HRHD pts referred to our Institution between January 2004 and June 2012. Results: Indication to HCT was given to 611 pts (median age 49 y, range 10-76; male 394), 120 pts (20%) received a transplant from a MRD; 187 pts (30%) activated a MUD search; 118 pts (19% of total pts, 63% of MUD searching) received a MUD transplant; 12 pts (2%) received a umbilical cord blood unit. Overall, 237 pts received a haplo-HCT (49%). The median time from indication to HCT was 87 days. The median time from indication to activation was 12 days and the median time from activation to HCT was 114 days (range 22-824). Lack of donor was not a limiting factor. Age was not a limiting variable to HCT execution: 107 patients (out of 487 SCT e 22%) were in the 60-69 range of age. The overall survival (OS) analysis in ITT for the entire populationwas 43% at 2 years, with a median time of survival of 767 days. The 2yOS in pts transplanted in CRwas 60%, in pts transplanted in PD is 25% (p CR1 and PD, respectively. The outcome analysis (OS, relapse incidence, transplant related mortality) per donor source was comparable (p1⁄4ns) within CR setting. Conclusion: For many pts with HRHD, HCT from a MRD provides the best chance for long-term survival. However, only approximately 30% of individuals have a MRD. In our experience, haplo-HCT offers a concrete option of cure to HRHD pts: outcome results are superimposable to MRD and MUD in pts in CR at HCT. In ITT analysis, 80% of candidate pts received an HCT as a potential immunotherapy. All patients affected by HRHD, for whom an HCT is part of their therapeutic program, should proceed with a well-timed HCT whatever the stem cell source, and in the absence of a fully HLA-compatible donor the option of a haploidentical donor should be simultaneously considered.