An Analysis of the Worldwide Utilization of Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia.

Acute myeloid leukemia (AML) has an aggressive course and a historically dismal prognosis. For many patients, hematopoietic stem cell transplantation (HSCT) represents the best option for cure, but access, utilization, and health inequities on a global scale remain poorly elucidated. We wanted to describe patterns of global HSCT use in AML for a better understanding of global access, practices, and unmet needs internationally. Estimates of AML incident cases in 2016 were obtained from the Global Burden of Disease 2019 study. HSCT activities were collected from 2009 to 2016 by the Worldwide Network for Blood and Marrow Transplantation through its member organizations. The primary endpoint was global and regional use (number of HSCT) and utilization of HSCT (number of HSCT/number of incident cases) for AML. Secondary outcomes included trends from 2009 to 2016 in donor type, stem cell source, and remission status at time of HSCT. Global AML incidence has steadily increased, from 102,000 (95% uncertainty interval: 90,200-108,000) in 2009 to 118,000 (104,000-126,000) in 2016 (16.2%). Over the same period, a 54.9% increase from 9659 to 14,965 HSCT/yr was observed globally, driven by an increase in allogeneic (64.9%) with a reduction in autologous (-34.9%) HSCT. Although the highest numbers of HSCT continue to be performed in high-resource regions, the largest increases were seen in resource-constrained regions (94.6% in Africa/East Mediterranean Region [AFR/EMR]; 34.7% in America-Nord Region [AMR-N]). HSCT utilization was skewed toward high-resource regions (in 2016: AMR-N 18.4%, Europe [EUR] 17.9%, South-East Asia/Western Pacific Region [SEAR/WPR] 11.7%, America-South Region [AMR-S] 4.5%, and AFR/EMR 2.8%). For patients <70 years of age, this difference in utilization was widened; AMR-N had the highest allogeneic utilization rate, increasing from 2009 to 2016 (30.6% to 39.9%) with continued low utilization observed in AFR/EMR (1.7% to 2.9%) and AMR-S (3.5% to 5.4%). Across all regions, total HSCT for AML in first complete remission (CR1) increased (from 44.1% to 59.0%). Patterns of donor stem cell source from related versus unrelated donors varied widely by geographic region. SEAR/WPR had a 130.2% increase in related donors from 2009 to 2016, and >95% HSCT donors in AFR/EMR were related; in comparison, AMR-N and EUR have a predilection for unrelated HSCT. Globally, the allogeneic HSCT stem cell source was predominantly peripheral blood (69.7% of total HSCT in 2009 increased to 78.6% in 2016). Autologous HSCT decreased in all regions from 2009 to 2016 except in SEAR/WPR (18.9%). HSCT remains a central curative treatment modality in AML. Allogeneic HSCT for AML is rising globally, but there are marked variations in regional utilization and practices, including types of graft source. Resource-constrained regions have the largest growth in HSCT use, but utilization rates remain low, with a predilection for familial-related donor sources and are typically offered in CR1. Further studies are necessary to elucidate the reasons, including economic factors, to understand and address these health inequalities and improve discrepancies in use of HSCT as a potentially curative treatment globally.

Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Children with High-Risk Leukemia Using a Reduced-Intensity Conditioning Regimen and Peripheral Blood as the Stem Cell Source.

The use of haploidentical donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide (Haplo-PTCy) in children is increasing; however, it is still not clear which preparative regimen is best in this setting. We present the long-term results of 42 patients age <18 years with high-risk leukemia who underwent this procedure using a reduced-intensity conditioning regimen (RIC) and peripheral blood as the stem cell source. Twenty-six patients had acute lymphoblastic leukemia, 13 had acute myelogenous leukemia, 2 had juvenile myelomonocytic leukemia, and 1 had blast crisis of chronic myelogenous leukemia. One-third of the patients were in first remission, 50% were in second remission, 14% were in third remission, and 3% had refractory disease. Neutrophil recovery occurred in 100% of the 40 patients alive at day +30, and transplantation-related mortality at 1 year was 14%. The incidence of acute graft-versus-disease (GVHD) grade III-IV was 17%, and the cumulative incidence of moderate to severe chronic GVHD at 1 year was 29%. The median duration of follow-up for surviving patients was 45 months; overall survival and event-free survival at 36 months were 56% and 46%, respectively. Long-term results of this series show that the use of an RIC regimen with peripheral blood stem cells as the cell source, in children with high-risk leukemia who underwent haplo-PTCy has tolerable toxicity, universal engraftment, and good survival rates.

Policitemia secundaria a nefrocalcitosis y acidosis tubular renal / Secondary Polycithemia and nephrocalcynosis and renal tubular acidosis

Las principales causas de policitemia secundaria son las enfermedades pulmonares y cardíacas productoras de hipoxia tisular. Sin embargo, en muy raras ocasiones, es el riñon el causante de la eritrocitosis. Las patologías renales que se asocian con mayor frecuencia a este fenómeno son los riñones poliquísticos, los quistes simples, el hipernefroma, la hidronefrosis, el cistadenoma y la estenosis de la arteria renal (2). No obstante, hay informados en la literatura en inglés cerca de 12 casos en los que la causa fue una nefrocalcinosis. A continuación presentamos un caso visto en el Hospital San Vicente de Paúl de Medellín con esta muy rara asociación.