Data on 68 146 hematopoietic stem cell transplants (HSCTs) (53% autologous and 47% allogeneic) gathered by 1566 teams from 77 countries and reported through their regional transplant organizations ...were analyzed by main indication, donor type and stem cell source for the year 2012. With transplant rates ranging from 0.1 to 1001 per 10 million inhabitants, more HSCTs were registered from unrelated 16 433 donors than related 15 493 donors. Grafts were collected from peripheral blood (66%), bone marrow (24%; mainly non-malignant disorders) and cord blood (10%). Compared with 2006, an increase of 46% total (57% allogeneic and 38% autologous) was observed. Growth was due to an increase in reporting teams (18%) and median transplant activity/team (from 38 to 48 HSCTs/team). An increase of 167% was noted in mismatched/haploidentical family HSCT. A Strengths, Weaknesses, Opportunities, Threats (SWOT) analysis revealed the global perspective of WBMT to be its major strength and identified potential to be the key professional body for patients and authorities. The limited data collection remains its major weakness and threat. In conclusion, global HSCT grows over the years without plateauing (allogeneic>autologous) and at different rates in the four World Health Organization regions. Major increases were observed in allogeneic, haploidentical HSCT and, to a lesser extent, in cord blood transplantation.
Advances in hematopoietic cell transplantation (HCT) technology and supportive care techniques have led to improvements in long-term survival after HCT. Emerging indications for transplantation, ...introduction of newer graft sources (for example, umbilical cord blood) and transplantation of older patients using less intense conditioning regimens have also contributed to an increase in the number of HCT survivors. These survivors are at risk for developing late complications secondary to pre-, peri- and post-transplant exposures and risk factors. Guidelines for screening and preventive practices for HCT survivors were published in 2006. An international group of transplant experts was convened in 2011 to review contemporary literature and update the recommendations while considering the changing practice of transplantation and international applicability of these guidelines. This report provides the updated recommendations for screening and preventive practices for pediatric and adult survivors of autologous and allogeneic HCT.
Haematopoietic cell transplants are done by more than 1500 transplant centres in 75 countries, mostly for life-threatening haematological disorders. However, transplant technology and access are not ...uniformly distributed worldwide. Most transplants are done predominately in Europe, North America and some Asian countries. We review transplant activity in Latin America, a geographic region with a population of >600 million persons living in countries with diverse economic and social development levels. These data indicate a 20-40-fold lower frequency of transplants in Latin America compared with Europe and North America. We show that although economics, infrastructure and expertise are important limitations, other variables also operate. Changes in several of these variables may substantially increase transplant activity in Latin America.
Invasive fungal disease (IFD) shows distinct regional incidence patterns and epidemiological features depending on the geographic region. We conducted a prospective survey in eight centres in Brazil ...from May 2007 to July 2009. All haematopoietic cell transplant (HCT) recipients and patients with acute myeloid leukaemia (AML) or myelodysplasia (MDS) were followed from admission until 1 year (HCT) or end of consolidation therapy (AML/MDS). The 12-month cumulative incidence (CI) of proven or probable IFD was calculated, and curves were compared using the Grey test. Among 237 AML/MDS patients and 700 HCT recipients (378 allogeneic, 322 autologous), the 1-year CI of IFD in AML/MDS, allogeneic HCT and autologous HCT was 18.7%, 11.3% and 1.9% (p <0.001), respectively. Fusariosis (23 episodes), aspergillosis (20 episodes) and candidiasis (11 episodes) were the most frequent IFD. The 1-year CI of aspergillosis and fusariosis in AML/MDS, allogeneic HCT and autologous HCT were 13.4%, 2.3% and 0% (p <0.001), and 5.2%, 3.8% and 0.6% (p 0.01), respectively. The 6-week probability of survival was 53%, and was lower in cases of fusariosis (41%). We observed a high burden of IFD and a high incidence and mortality for fusariosis in this first multicentre epidemiological study of IFD in haematological patients in Brazil.
The literature describing methods for estimating animal abundance and related parameters continues to grow. This paper reviews recent developments in the subject over the past seven years and updates ...two previous reviews.
Metabolic syndrome (MetS) is a constellation of cardiovascular risk factors that increases the risk of cardiovascular disease, diabetes mellitus and all cause mortality. Long-term survivors of ...hematopoietic cell transplantation (HCT) have a substantial risk of developing MetS and cardiovascular disease, with the estimated prevalence of MetS being 31-49% among HCT recipients. Although MetS has not yet been proven to impact cardiovascular risk after HCT, an understanding of the incidence and risk factors for MetS in HCT recipients can provide the foundation to evaluate screening guidelines and develop interventions that may mitigate cardiovascular-related mortality. A working group was established through the Center for International Blood and Marrow Transplant Research and the European Group for Blood and Marrow Transplantation with the goal of reviewing literature and recommend practices appropriate to HCT recipients. Here we deliver consensus recommendations to help clinicians provide screening and preventive care for MetS and cardiovascular disease among HCT recipients. All HCT survivors should be advised of the risks of MetS and encouraged to undergo recommended screening based on their predisposition and ongoing risk factors.
Haploidentical hematopoietic-cell transplantation using post-transplant cyclophosphamide(Haplo-PTCy) is a feasible procedure in children with haematologic malignancies. However, data of a large ...series of children with acute leukaemia(AL) in this setting is missing. We analysed 144 AL Haplo-PTCy paediatric recipients; median age was 10 years. Patients had acute lymphoblastic(ALL; n = 86) or myeloblastic leukaemia(AML; n = 58) and were transplanted in remission(CR1: n = 40; CR2: n = 57; CR3+: n = 27) or relapse (n = 20). Bone marrow was the graft source in 57%; donors were father (54%), mother (35%), or sibling (11%). Myeloablative conditioning was used in 87%. Median follow-up was 31 months. At day +100, cumulative incidence (CI) of neutrophil recovery and acute GVHD (II-IV) were 94% and 40%, respectively. At 2-years, CI of chronic GVHD and relapse, were 31%, 40%, and estimated 2-year overall survival (OS), leukaemia-free survival (LFS) and graft-versus-host-relapse-free survival (GRFS) were 52%, 44% and 34% respectively. For patients transplanted in remission, positive measurable residual disease (MRD) prior to transplant was associated with decreased LFS (p = 0.05) and GRFS (p = 0.003) and increased risk of relapse (p = 0.02). Mother donor was associated with increased risk of chronic GVHD (p = 0.001), decreased OS (p = 0.03) and GRFS (p = 0.004). Use of PBSC was associated with increased risk of chronic GVHD (p = 0.04). In conclusion, achieving MRD negativity pre-transplant, avoiding use of mother donors and PBSC as graft source may improve outcomes of Haplo-PTCy in children with AL.
Haematopoietic cell transplant activity in the 28 countries comprising Latin America is poorly defined. We conducted a voluntary survey of members of the Latin American Bone Marrow Transplantation ...Group regarding transplant activity 2009-2012. Collated responses were compared with data of transplant rates from the Worldwide Network for Blood and Marrow Transplantation for other geographic regions. Several socio-economic variables were analysed to determine correlations with transplant rates. In total, 94 teams from 12 countries reported 11 519 transplants including 7033 autotransplants and 4486 allotransplants. Annual activity increased from 2517 transplants in 2009 to 3263 in 2012, a 30% increase. Median transplants rate (transplant per million inhabitants) in 2012 was 64 (autotransplants, median 40; allotransplants, median 24). This rate is substantially lower than that in North America and European regions (482 and 378) but higher than that in the Eastern Mediterranean and Asia Pacific regions (30 and 45). However, the Latin America transplant rate is 5-8-fold lower than that in America and Europe, suggesting a need to increase transplant availability. Transplant team density in Latin America (teams per million population; 1.8) is 3-4-fold lower than that in North America (6.2) or Europe (7.6). Within Latin America, there is substantial diversity in transplant rates by country partially explained by diverse socio-economic variables including per capita gross national income, health expenditure and physician density. These data should help inform future health-care policy in Latin America.
Introduction: Relapse of a malignant hematological disease after allogeneic HCT is associated with poor survival and may not be treated with a curative intent. However, a second HCT (HCT2) may ...achieve durable remission. Objective: To determine the outcomes of pediatric patients who received a HCT2 for relapsed malignant hematological diseases. Casuistic and method: Review of the medical records of pediatric patients who underwent a HCT2 for relapsed malignant hematological diseases in two institutions from 2013 to 2023. OS was estimated using Kaplan-Meier survival analysis. The conventional HCT Comorbidity index (HCT-CI) was calculated for all patients (http://www.hctci.org/Home/Calculator) categorized in < or ≥ 2. Results: Nineteen patients with a median age of 10 years (range, 2–17) underwent HCT2 for B-ALL (n = 8), T-ALL (n = 2), AML (n = 5), CML-BC (n = 2), MDS (n = 1) and JMML (n = 1). Donor types were unrelated (n = 4), haploidentical (n = 11), and unrelated cord-blood (n = 4). Donors were different at HCT2 in all patients and all haploidentical HCT2 used the other haplotype. All nineteen patients received myeloablative conditioning, and 52% (n = 10) were in remission at HCT2. The median remission duration after HCT1 was 12 months (range, 2–22) and the median time between transplants was 16 months (range, 5–30). The median follow-up of surviving patients after HCT2 was 33 months (range, 9–117), with 47% alive at time of analysis. The most common cause of death was disease recurrence (n = 6, 31%). At time of analysis, OS, PFS, relapse, and Transplant-Related Mortality (TRM) were 47% , 42%, 45%, and 21%, respectively. OS was 37% (3/8) for B-ALL. All T-cell ALL, CML-BC, MDS and JMML patients are alive, but all 5 patients with AML have died. None of the latter have used post-HCT maintenance to prevent relapse. Pre-HCT2 remission status did not appear to influence OS and PFS, since of 11 patients in remission, 5 remain alive and disease-free. Of the 8 patients transplanted with active disease, 6 remain in remission. Ten patients had HCT-CI < 2 pre HCT2, and 3 of 10 have died. However, 7 out of 9 patients with HCT-CI score ≥ 2 died (HR = 4.8, p = 0.007). Conclusion: A second HCT can be feasible for patients with relapsed malignant hematological diseases. Overall survival is higher than 40%, suggesting that this approach may cure a proportion of the patients, particularly those with HCT-CI < 2 pre HCT2.
Introduction: Acute Promyelocytic Leukemia (APL) has a high early morbidity and mortality, but a high cure rate. Primary refractory or relapsed disease are referred to HSCT. Patients with molecular ...remission undergo autologous, and allogeneic HSCT is reserved for those with molecular resistance/persistence or multiple relapses. It is the only pediatric leukemia for which autologous HSCT is indicated, as it is believed that there is no significant Graft-Versus-Leukemia (GVL) effect. There is scarce information on the outcome of HSCT for pediatric APL, and none to our knowledge in Brazil. Objective: To review the results of autologous and allogeneic HSCT performed for the treatment of pediatric APL. Method: retrospective analysis of the medical records of all patients with APL referred to HSCT. Results: A total of 121 HSCT were performed for AML between 2003 and 2023, 15 of them for APL. Three patients had Central Nervous System (CNS) disease prior to HSCT. The median age was 13.6 years (7.8‒21.7); 27% were female. Three patients were transplanted in 1st remission due to prior molecular persistent disease; 8 in second remission, and 4 in very advanced disease.Ten patients underwent autologous HSCT, 7 of them after arsenic (ATO)-based therapy; 9 had Busulfan-based conditioning and 1 TBI-based due to CNS relapse. With a median follow-up of 126 months, all patients were alive. Two patients received ATO post autologous HSCT, one due to molecular relapse and the second due to disease detected by PCR in the graft.Five patients underwent allogeneic HSCT due to very advanced disease. Donors were HLA-identical related (3), MUD (1) and haploidentical (1); ATO was used pre HSCT in two of them. All patients were in morphological remission, but one had persistent molecular disease. Three patients had Busulfan-based conditioning and 2 TBI. Three are alive with a median follow-up of 117 months. Conclusion: in this retrospective cohort, both autologous and allogeneic transplantation were effective therapies. All 10 patients are alive after autologous HSCT. Three of 5 patients with very advanced disease are alive and disease and GVHD-free.
PDF
