Investigation of mitochondrial stress inducers on human bone marrow derived mesenchymal stem cells for mitochondrial disease model

42nd Congress of the Federation-of-European-Biochemical-Societies (FEBS) on From Molecules to Cells and Back -- SEP 10-14, 2017 -- Jerusalem, ISRAELWOS: 000409918902099…Federat European Biochem So

Initial white blood cell kinetics for assessment of individual response to the conditioning regimen in pediatric hematopoietic stem cell transplantation patients.

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Incidence and outcome of Kaposi sarcoma after hematopoietic stem cell transplantation: a retrospective analysis and a review of the literature, on behalf of infectious diseases working party of EBMT

The incidence, the clinical characteristics, and the outcome of Kaposi sarcoma (KS) in patients after hematopoietic stem cell transplantation (HSCT) were assessed. During the period 1987\u20132018, 13 cases of KS were diagnosed, 3 females and 10 males, median age of 50 years, median time from HSCT of 7 months. KS had an incidence of 0.17% in allogeneic and 0.05% in autologous HSCT. HHV-8 was documented in eight of nine tumor tissue samples assessed. The organ involvement was: skin in nine, lymph nodes in six, oral cavity in four, and visceral in three patients, respectively; seven patients had >1 organ involved. Five patients had immunosuppression withdrawn, whereas four and three patients received radiotherapy and chemotherapy, respectively. Eight patients are alive (median follow-up 48 months, range 5\u2013128), whereas five patients died after a median time of 8 months from the diagnosis of KS. However, no death was caused by KS. We conclude that the incidence of KS after HSCT is very low. Although KS can be managed with the reduction of immunosuppression, visceral forms may require chemotherapy and/or radiotherapy. The low prevalence of KS indicates that screening for HHV-8 serology and surveillance for HHV-8 viremia are not indicated in HSCT patients

Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: A retrospective analysis on behalf of <scp>EBMT</scp> group

AbstractAllogeneic hematopoietic stem cell transplantation (allo‐HSCT) is curative for bone marrow failure in patients with Fanconi anemia (FA), but the presence of a malignant transformation is associated with a poor prognosis and the management of these patients is still challenging. We analyzed outcome of 74 FA patients with a diagnosis of myelodysplastic syndrome (n = 35), acute leukemia (n = 35) or with cytogenetic abnormalities (n = 4), who underwent allo‐HSCT from 1999 to 2016 in EBMT network. Type of diagnosis, pre‐HSCT cytoreductive therapies and related toxicities, disease status pre‐HSCT, donor type, and conditioning regimen were considered as main variables potentially influencing outcome. The 5‐year OS and EFS were 42% (30‐53%) and 39% (27‐51%), respectively. Patients transplanted in CR showed better OS compared with those transplanted in presence of an active malignant disease (OS:71%[48‐95] vs 37% [24‐50],P = .04), while none of the other variables considered had an impact. Twenty‐two patients received pre‐HSCT cytoreduction and 9/22 showed a grade 3‐4 toxicity, without any lethal event or negative influence on survival after HSCT(OS:toxicity pre‐HSCT 48% [20‐75%] vs no‐toxicity 51% [25‐78%],P = .98). The cumulative incidence of day‐100 grade II‐IV a‐GvHD and of 5‐year c‐GvHD were 38% (26‐50%) and 40% (28‐52%). Non‐relapse‐related mortality and incidence of relapse at 5‐years were 40% (29‐52%) and 21% (11‐30%) respectively, without any significant impact of the tested variables. Causes of death were transplant‐related events in most patients (34 out of the 42 deaths, 81%). This analysis confirms the poor outcome of transformed FA patients and identifies the importance of achieving CR pre‐HSCT, suggesting that, in a newly diagnosed transformed FA patient, a cytoreductive approach pre‐HSCT should be considered if a donor have been secured

Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

Allogeneic hematopoietic stem cell transplantation (HSCT) is the gold standard curative therapy for infants and children with many inborn errors of immunity (IEI), but adolescents and adults with IEI are rarely referred for transplant. Lack of published HSCT outcome data outside small, single-center studies and perceived high risk of transplant-related mortality have delayed the adoption of HSCT for IEI patients presenting or developing significant organ damage later in life. This large retrospective, multicenter HSCT outcome study reports on 329 IEI patients (age range, 15-62.5 years at HSCT). Patients underwent first HSCT between 2000 and 2019. Primary endpoints were overall survival (OS) and event-free survival (EFS). We also evaluated the influence of IEI-subgroup and IEI-specific risk factors at HSCT, including infections, bronchiectasis, colitis, malignancy, inflammatory lung disease, splenectomy, hepatic dysfunction, and systemic immunosuppression. At a median follow-up of 44.3 months, the estimated OS at 1 and 5 years post-HSCT for all patients was 78% and 71%, and EFS was 65% and 62%, respectively, with low rates of severe acute (8%) or extensive chronic (7%) graft-versus-host disease. On univariate analysis, OS and EFS were inferior in patients with primary antibody deficiency, bronchiectasis, prior splenectomy, hepatic comorbidity, and higher hematopoietic cell transplant comorbidity index scores. On multivariable analysis, EFS was inferior in those with a higher number of IEI-associated complications. Neither age nor donor had a significant effect on OS or EFS. We have identified age-independent risk factors for adverse outcome, providing much needed evidence to identify which patients are most likely to benefit from HSCT.Transplantation and immunomodulatio