JOURNAL OF BONE MARROW TRANSPLANTATION AND CELLULAR THERAPY
The Journal of Bone Marrow Transplantation and Cellular Therapy (JBMTCT) was established in 2020 as the official vehicle for disseminating scientific information from the Brazilian Society of Bone Marrow Transplantation (Sociedade Brasileira de Terapia Celular e Transplante de Medula Óssea - SBTMO). The target audience is researchers, teachers, professionals and students with an interest in the medicine field, especially bone marrow transplantation, cell therapy and related sciences. Aiming to facilitate the immediate dissemination of articles, the Journal of Bone Marrow Transplantation and Cellular Therapy adopts a continuous publication model, with articles made available as soon as they are approved and are compiled into a single annual volume. The journal's abbreviated title is J Bone Marrow Transplant Cell Ther and should be used in bibliographies, footnotes, references and bibliographic captions. Mission To promote scientific advancement and clinical innovation in hematopoietic stem cell transplantation and advanced cell therapies through the publication of high-impact original research, critical reviews, practical reports, protocols, pediatric and adult consensus updates, and cell therapy manuals. The journal seeks to connect researchers, hematologists, oncologists, and multidisciplinary teams globally, driving patient safety, treatment efficacy, and the development of new frontiers in the field. Focus and Scope The Journal of Bone Marrow Transplantation and Cellular Therapy publishes the results of basic and applied research that promotes scientific development in Hematology, Bone Marrow Transplantation, Cellular Therapy and related áreas. Fees The Journal of Bone Marrow Transplantation and Cellular Therapy does not charge fees for submission or processing of articles. Open Access The Journal of Bone Marrow Transplantation and Cellular Therapy follows the Diamond Open Access model. The Journal of Bone Marrow Transplantation and Cellular Therapy offers immediate open access to its content, adhering to the principles of free access and the democratization of scientific knowledge. All content is freely available without charge to the user or his/her institution. Users are allowed to read, download, copy, distribute, print, search, or link to the full texts of the articles, or use them for any other lawful purpose, without asking prior permission from the publisher or the author, including commercial purposes, provided that proper credit is given. To maximize the dissemination and use of published material, the Journal of Bone Marrow Transplantation and Cellular Therapy adopts the Creative Commons license with CC BY 4.0 attribution. Also, the copyright belongs to the authors. Authors are allowed to deposit all versions of their work [Preprint (submitted version), Accepted Manuscript (version of the manuscript that received acceptance), Published Article (version of record)] in an institutional or thematic repository without embargo. Ethics in Publication The publication involves the authors, the editors, the reviewers, and the Brazilian Society of Bone Marrow Transplantation who have responsibilities that affect all process stages, from submission to publication. The Journal of Bone Marrow Transplantation and Cellular Therapy is committed to high standards of ethical behavior in management and editorial practices, with an emphasis on relationships with authors and, mainly, in the evaluation of their manuscripts, under procedures established by international institutions such as the Committee on Publication Ethics (COPE) Principles of Transparency and Best Practice in Scholarly Publishing), Directory of Open Access Journals (DOAJ) (Transparency & Best Practice), Open Access Scholarly Publishing Association (OASPA) (Principles of Transparency and Best Practice in Scholarly Publishing), Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP) (Code of Good Scientific Practices) e Scientific Electronic Library Online (SciELO) Guidelines on Best Practices for Strengthening Ethics in Scientific Publication). The Editorial Board ensure the quality of the material they publish, prohibiting any form of plagiarism and/or fraudulent data to maintain the integrity of the academic record and ensure intellectual standards. They are always ready to publish corrections, clarifications and retractions, when necessary, thus following the Guidelines for Good Practices in Scientific Publishing. The Journal of Bone Marrow Transplantation and Cellular Therapy adheres to international guidelines for good practice, recommended principles for investigating cases of research misconduct, and texts suggested by the World Science Forum and the Council of Scientific Editors when conducting investigations into international misconduct. Digital Preservation The Journal of Bone Marrow Transplantation and Cellular Therapy utilizes the PKP PN system to establish a distributed archiving system among participating libraries, enabling them to create permanent journal archives for preservation and future access. Archiving The Journal of Bone Marrow Transplantation and Cellular Therapy also uses the system LOCKSS for the preservation and archiving of the issues. Updated in April, 2026.
Sociedade Brasileira de Transplante de Medula Óssea
Objective: To describe the main knowledge and skills required of pharmacists who work or wish to work in chimeric antigen receptor T cells (CAR-T) cell therapy. Methods: This is a narrative literature review based on international guidelines, clinical trials, and recommendations from specialized societies in cellular therapy and oncology, focusing on pharmaceutical practice. Results: CAR-T cell therapy has emerged as a transformative treatment for patients with relapsed or refractory hematologic malignancies. Pharmacists play a critical role throughout the care continuum, including patient eligibility assessment, support during lymphodepletion regimens, and management of cellular product logistics, such as storage and traceability. Clinical pharmacists are also essential in the early recognition and management of adverse events, particularly cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome, including the appropriate use of tocilizumab and corticosteroids. Additionally, medication reconciliation, drug interaction assessment, and education of healthcare teams and patients are key responsibilities. Conclusion: The involvement of pharmacists in CAR-T therapy is crucial to ensure patient safety and optimize clinical outcomes, requiring specialized training and strong integration within the multidisciplinary team.
Copyright (c) 2026 Daniella Cristina Oliveira, Jenifer Freitas da Silva https://creativecommons.org/licenses/by/4.0
Regulatory and operational framework for Chimeric Antigen Receptor T cell therapy in Brazil
Objective: Provides an overview of Brazil’s CAR-T regulatory structure, emphasizing post-authorization surveillance, clinical approval, manufacturing, and product release. Methods: Good Manufacturing Practice, ethics, biosafety, quality control, traceability, pharmacovigilance, and international harmonization are among the national and ANVISA laws governing advanced therapeutic products reviewed in this work. Results: Brazilian CAR-T manufacturing requires GMP-certified facilities, trained workers, and proven processes in addition to quality control and environmental monitoring. ANVISA and CONEP approval, as well as CTNBio authorization for GMOs, are necessary for clinical investigations. Sterility, endotoxin levels, mycoplasma presence, viability, potency, vector copy number, transduction efficiency, identification, and traceability are some of the variables that affect the product release process. Pharmacovigilance, safety reporting, inspections, and manufacturing management are among the duties that follow approval. Conclusion: Brazil has established a comprehensive regulatory framework for CAR-T therapy that accounts for both regional operational requirements and international norms. Expanding safe, long-term access to cell and gene therapies would require bolstering national manufacturing capacity and the development of flexible regulatory frameworks.
Copyright (c) 2026 Andrea Kondo, Felipe Mesquita, Léo Sekine, Marília Maia, Nelson Hamerschlak https://creativecommons.org/licenses/by/4.0
Chimeric antigen receptor-natural killer
Chimeric antigen receptor (CAR) technology has transformed adoptive cell therapy, particularly through the success of CAR-T cells in hematologic malignancies. However, challenges such as toxicity, complex manufacturing, and high costs have encouraged the exploration of alternative immune cell platforms. Natural killer (NK) cells represent a promising approach due to their intrinsic cytotoxic activity, major histocompatibility complex-independent tumor recognition, and lower risk of severe immune-related toxicities. CAR-NK cells combine CAR-mediated antigen targeting with the natural antitumor functions of NK cells, supporting the development of safer and potentially “off-the-shelf” cellular therapies. This chapter reviews the biological rationale of CAR-NK cells, current gene engineering strategies, and the evolving clinical landscape, highlighting their potential in hematologic malignancies, solid tumors, and autoimmune diseases.
Copyright (c) 2026 Larissa Cardoso Zanetti, Raquel de Melo Alves Paiva, Lucila Nassif Kerbauy https://creativecommons.org/licenses/by/4.0
Lymphocyte collection for cellular therapy
Introduction: Leukapheresis is the starting point of cellular therapies such as chimeric antigen receptor T cells (CAR-T cells) and donor lymphocyte infusion. Methods: After a review of the literature, we provided guidance on how to evaluate candidates for leukapheresis and how to optimize the safety and efficacy of the procedure. Results: Clinical and laboratory assessment is warranted prior to leukapheresis. T cell fitness is impacted by previous treatments, ageing, and tumor burden. The most common adverse reaction is hypocalcemia. Conclusion: Leukapheresis is a safe and effective procedure. Technical and clinical aspects must be observed to preserve T cell fitness, ensure manufacturing success, and guarantee patient safety and clinical efficacy. The current challenge is to standardize guidelines for pre-leukapheresis requirements and collection yield improvement.
Copyright (c) 2026 Sérgio Luiz Arruda Parente Filho, Suzanna Tavares Barbosa, Naliele Cristina Maia de Castro, Araci Sakashita https://creativecommons.org/licenses/by/4.0
Genetic engineering strategies for cell-based therapy: viral, non-viral, and precision genome editing technologies
Objective: To review the existing genetic engineering strategies commonly used for generating chimeric antigen receptor (CAR)-T cell therapy. Methods: This article provides a comprehensive literature review and compares different approaches on viral, non-viral and precision genome editing technologies, aiming to provide an overview and guidelines for methods selection during clinical CAR-T cell manufacturing. Results: CAR-T cell therapy has revolutionized cancer treatment for hematological malignancies. The manufacturing of CAR-T cell therapy relies on the genetic modification of T cells to achieve ectopic CAR gene expression. Depending on the evolving CAR designs, viral, non-viral, and genome editing platforms are used for optimal CAR expression, which could determine the clinical efficacy and safety profiles of these products. Efforts have been ongoing to empower CAR-T cell efficacy while minimizing toxicity over the past decades. Conclusion: Successful CAR-T cell therapy depends on rational platform selection and optimization of gene delivery methods based on clinical needs and context.
Copyright (c) 2026 Wing Keung Chan, Marcos de Lima https://creativecommons.org/licenses/by/4.0
Current use and outcomes of cellular therapy: Brazilian Summary Slides – 2026
Objective: To describe CAR-T cell therapy activity and outcomes reported by Brazilian centers participating in the Brazilian Society of Cellular Therapy and Bone Marrow Transplantation (SBTMO)/Center for International Blood and Marrow Transplant Research (CIBMTR) network between January 2020 and December 2025. Methods: Data from patients who received commercial or academic CAR-T cell therapies in Brazilian centers were extracted from the CIBMTR database. Participating centers reported data through the FormsNet3 platform, allowing standardized collection of patient, disease, and treatment-related variables. Descriptive analyses were performed, and overall survival (OS) was estimated using the Kaplan–Meier method. Results: A total of 219 CAR-T cell infusions were reported. The median age was 57 years (range, 4–83 years). The most common indications were non Hodgkin lymphoma (NHL) (146 patients, 67%), multiple myeloma (MM) (39 patients, 18%), acute lymphoblastic leukemia (ALL) (32 patients, 14%), and chronic lymphocytic leukemia (CLL) (2 patients, 1%). Commercial CAR-T products accounted for 89% of all treatments and were predominantly used for NHL (n = 135) and MM (n = 35). Most deaths were attributed to disease progression or relapse (n = 42), followed by infections (n = 14). After a median followup of 365 days, the 1-year OS was 63% (95% CI, 54–73%) among patients with NHL and 73% (95% CI, 57–93%) among patients with ALL. Conclusion: This report documents the implementation and rapid expansion of CAR-T cell therapy in Brazil among centers reporting to the SBTMO/CIBMTR network. The continued development of this collaborative infrastructure supports national monitoring of CAR-T activity, facilitates outcome assessment, and contributes to quality assurance, benchmarking initiatives, and compliance with national and international regulatory requirements.
Copyright (c) 2026 Cinthya Corrêa da Silva, Anderson João Simione, Giancarlo Fatobene, Jayr Schmidt Filho, Adriana Seber, Nelson Hamerschlak, João Samuel de Holanda Farias, Vaneuza Araújo Moreira Funke, Phillip Scheinberg, Maria Claudia Rodrigues Moreira, Abel da Costa Neto, Vinicius Campos de Molla, Cilmara Cristina Kuwahara Dumke , Yana Augusta Sarkis Novis, Vanderson Rocha, Fernando Sérgio Blumm Ferreira, Volney Assis Lara Vilela, Grazielly Vargas Grilo, Andresa Lima Melo, Andreza Alice Feitosa Ribeiro, Marcos Paulo Colella, Afonso Celso Vigorito, Marcelo C. Pasquini, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Current activity trends and outcomes of hematopoietic cell transplantation: A 2026 report from the Brazilian Registry of Hematopoietic Cell Transplantation in collaboration with the CIBMTR & SBTMO
The development of the Brazilian Registry of Hematopoietic Cell Transplantation (Registro Brasileiro de Transplantes de Células Hematopoieticas), in collaboration with the Center for International Blood and Marrow Transplant Research (CIBMTR), continues to provide a comprehensive assessment of hematopoietic cell transplantation (HCT) activity and outcomes in Brazil. In this study, we report an updated analysis of national transplant activity. Brazilian transplant centers report their data to the CIBMTR using the FormsNet3 platform, and data are returned to the Brazilian Society of Cellular Therapy and Bone Marrow Transplantation (Sociedade Brasileira de Terapia Celular e Transplante de Medula Óssea) through the Data Back to Centers (DBtC) tool. Data from patients who underwent HCT from 2015 to 2025 in Brazilian centers were extracted from the CIBMTR. Descriptive analyses were performed using patient-, disease-, and transplant-related variables, and overall survival was estimated using the Kaplan–Meier method. A total of 15,586 HCTs were included (7,507 autologous and 8,079 allogeneic). For survival analyses, 9,150 patients who underwent a first HCT from 2015 to 2023 were analyzed, with a median follow-up of 35 months. The number of reporting centers increased over time, reaching 51 centers during the study period. Acute leukemias remained the most common indication for allogeneic HCT, while multiple myeloma and lymphomas predominated in autologous HCT. In recent years, mismatched related donors have accounted for the largest proportion of allogeneic transplants. Bone marrow was the main graft source in pediatric patients, whereas peripheral blood predominated in adults. Infections were the most frequent cause of death within the first 100 days after HCT, while primary disease was the leading cause beyond 100 days. Survival patterns varied according to disease characteristics and transplantrelated factors, with lower survival estimates observed in patients transplanted with advanced disease stages. Comparisons with the United States Summary Slides suggest both similarities and differences in transplant activity and outcomes, which should be interpreted with caution given variations in data structure, patient characteristics, and risk stratification.
Maria Cristina Martins de Almeida Macedo
Copyright (c) 2026 Anderson João Simione, Cinthya Corrêa da Silva, Afonso Celso Vigorito, Antonio Vaz Macedo, Heliz Regina Alves das Neves, Vanessa Aparecida do Nascimento Varjão, Monique Ammi, Flavia Ferreira da da Costa, Adriana Mendes de Quadros Cavilha, Alayne Magalhães T.D. Yamada, Vergilio Antonio Rensi Colturato, Phillip Scheinberg, Samir Kanaan Nabhan, Decio Lerner, Nelson Hamerschlak, Marcos Paulo Colella, George Maurício Navarro Barros, Alexandre Silvério, Adriana Seber, Simone Ojima Ferreira, Yana Augusta Sarkis Novis, Andreia Ribeiro de Almeida, Vanderson Geraldo Rocha, Maria Claudia Rodrigues Moreira, Claudia Caceres Astigarraga, Liane Esteves Daudt, Maria Cristina Martins de Almeida Macedo, Ricardo Chiattone, Juliana Folloni Fernandes, Volney Assis Lara Vilela, Rodolfo Daniel de Almeida Soares, Gustavo Machado Teixeira, Celso Arrais-Rodrigues, Roberto Luiz da Silva, Vaneuza Araújo Moreira Funke, Jayr Schmidt Filho, Vinicius Campos de Molla, João Samuel de Holanda Farias, Ricardo Pasquini, Carmem Maria Sales Bonfim, Abrahão Elias Hallack Neto, Rodolfo Froes Calixto, João Victor Piccolo Feliciano, Rafael Dezen Gaiolla, Marcelo Capra, Angelo Atalla, Milton Alexandre Ferreira Aranha, Rony Schaffel, Gianne Donato Costa Veloso, Fernando Sergio Blumm Ferreira, Andresa Lima Melo, Simone de Castro Resende Franco, Marcelo Alves Aduan, Andreza Alice Feitosa Ribeiro, Thiago Xavier Carneiro, Wellington Moraes de Azevedo, Giancarlo Fatobene, Abel da Costa Neto, Marcelo C. Pasquini, Mary E. Flowers, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Effectiveness of therapeutic drug monitoring in patients undergoing allogeneic hematopoietic stem cell transplantation
Background: Therapeutic drug monitoring (TDM) aims to prevent excessive immunosuppression, graft rejection, and the emergence of graft-versus-host disease (GVHD) and to control drug interactions. Estimates suggest that acute GVHD (aGVHD) occurs in 30-50% of patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) and chronic GVHD (cGVHD) in 50 to 70%. Objective: To evaluate the effectiveness of TDM conducted by a clinical pharmacist on the quality of care for patients undergoing allogeneic HSCT to prevent GVHD and to improve clinical outcomes in comparison with historical controls. Methods: A cohort study of patients who underwent allogeneic HSCT was carried out from January 2015 to December 2017 (control group) and from January 2018 to December 2021 (TDM group). Patients were followed up for 2 years after HSCT. The exposed group received pharmaceutical TDM, considering the clinical pharmacokinetics of cyclosporine, tacrolimus, and busulfan, which was compared to a historical control group without TDM. Results: The control and TDM groups included 85 and 79 patients, respectively. Male patients predominated in this sample, the ages of whom ranged from 0 to 64 years. The incidence of GVHD in the overall cohort totaled 73.8% (n = 164). The incidence of aGVHD and cGVHD showed no significant difference between groups. Survival lasted for three more months (p = 0.04), and mortality was lower (risk ratio 0.55; p = 0.05) in the TDM group. Conclusion: TDM was associated with improved survival and reduced mortality, without significantly affecting the incidence of aGVHD or cGVHD.
Copyright (c) 2026 Joice Zuckermann, Bruno Mateus de Castro, Renata Garcia Ruschel, Lucas Bastos Beltrami, Rafaela Pivato da Fonseca, Priscila de Oliveira da Silva, Fernanda Fetter Scherer, Liane Esteves Daudt, Alessandra Aparecida Paz, Bibiana Verlindo de Araújo, Leila Beltrami Moreira https://creativecommons.org/licenses/by/4.0
The challenges in diagnosis and treatment of multiple myeloma in Latin America: a multinational survey and proposal for improvement
Multiple myeloma (MM) is a malignant hematologic disease marked by the abnormal growth of plasma cells, resulting in serious complications such as bone fractures, hypercalcemia, renal dysfunction, and anemia. It represents the second most frequent hematologic malignancy and is usually diagnosed at an advanced stage in Latin America. A multinational survey included 16 experts from 11 Latin American countries to share their experiences in myeloma healthcare, discuss existing gaps, and propose solutions. The public healthcare system struggles with slow diagnosis and treatment access, particularly in rural areas. The private system faces challenges related to insurance coverage and costs. A significant gap exists between the need for diagnostic tests and their availability. Access to new cancer treatments is hindered by inadequate public policies, high drug costs, and inconsistent approval processes. A comprehensive approach for improving MM management in Latin America is crucial, emphasizing training, access, stakeholder engagement, data enhancement, and financial resources.
Copyright (c) 2026 Camila Peña, Breno Gusmão , Eloisa Riva, Alana von Glasenapp, Dorotea Fantl, Rodrigo Meneces, Aline Ramírez, David Gomez Almaguer, Julio Dámaso Fernández-Aguila, Manolo Antônio Granja, Esmedaly Romero, Jule Vasquez, Humberto Martinez-Cordero , Ariel Corzo, Rafael Cunha, Fernanda Lemos Moura, Maria Victoria Mateos https://creativecommons.org/licenses/by/4.0
Outcomes of hematopoietic stem-cell transplantation in infants with KMT2A-rearranged acute lymphoblastic leukemia in first remission: a systematic review and single-arm meta-analysis
Infants with acute lymphoblastic leukemia (ALL) frequently experience poor outcomes, with KMT2A gene rearrangements (KMT2A-r) serving as the most significant adverse prognostic factor. Allogeneic hematopoietic stem cell transplantation (HSCT) in first complete remission (CR1) has been utilized to improve survival, yet its definitive role remains a subject of intense debate. This systematic review and meta-analysis aimed to evaluate survival outcomes of HSCT in infants with KMT2A-r ALL in CR1. We systematically searched PubMed, Embase, and the Cochrane Library for randomized and observational studies reporting survival outcomes for infants with KMT2A-r ALL undergoing HSCT in CR1. Survival data were synthesized using parametric modeling (Weibull distribution) to estimate pooled outcomes. Mean survival across 1,000 iterations per study was calculated with 95%CI. Heterogeneity was assessed using IÇ statistics. All analyses were performed using R software (version 4.1.3). Seven studies comprising 371 patients were included. The pooled analysis demonstrated a 4-year overall survival (OS) of 65% (95%CI 58.7-72.1), a 4-year disease-free survival of 57.5% (95%CI 51.8-64.3), and a 3-year event-free survival (EFS) of 53.2% (95%CI 41.5-66.1). Our findings suggest that HSCT in CR1 is a viable therapeutic strategy for selected high-risk infants with KMT2A-r ALL, showing improved survival compared to historical benchmarks. While novel and safer therapies are currently under investigation, HSCT remains a reasonable option. Clinical decisions should carefully balance the curative potential of transplantation against the risk of severe treatment-related toxicities and long-term sequelae.
Copyright (c) 2026 Bianca Lisa de Faria, Áurea Maria Salomão Simão, Pedro Henrique Aquino Gil de Freitas, Ana Beatriz Bertol, Amanda Silva Gomes, Barkhá Vijendra, Beatrice Araujo Duarte, Lilian Maria Cristofani https://creativecommons.org/licenses/by/4.0
Global expert perspectives on the future of hematopoietic cell transplantation and cell therapies
Settings: Hematopoietic cell transplantation (HCT) remains a cornerstone therapy for hematologic malignancies and several non-malignant disorders. At the same time, emerging therapies, particularly chimeric antigen receptor T cell (CAR-T) cells and other immunotherapies, are transforming the treatment landscape. Understanding how specialists anticipate these changes is critical for strategic planning in healthcare systems. Methodology: We conducted a global expert survey using a foresight informed framework to explore expected developments in HCT and related technologies over the next decade. Following a literature review of 4,312 publications indexed in Web of Science (2018–2022), a questionnaire was distributed to specialists in hematologic cancers and bone marrow transplantation identified through publication databases. Personalized email invitations were sent in April and May 2023. Descriptive analyses and non-parametric tests were used to examine differences according to self-reported knowledge levels. Results: A total of 1,711 valid responses were analyzed. Haploidentical transplantation was identified as the most promising transplantation modality for the next decade, particularly among respondents with higher knowledge levels. Severe combined immunodeficiency and sickle cell disease were the most frequently cited non-malignant indications. Knowledge gaps, regulatory barriers, and treatment costs were perceived as major obstacles to innovation, while limited health system infrastructure was identified as the main barrier to expanding access in middle-income countries. CAR-T cell therapy was considered the most promising emerging technology overall, although highly knowledgeable respondents more often identified bispecific antibodies as the most promising innovation. Conclusion: Experts anticipate continued growth in haploidentical transplantation alongside the expansion of advanced immunotherapies such as CAR-T cells. However, regulatory, financial, and infrastructure constraints may delay their widespread implementation, particularly in middle-income settings. Although several emerging therapies discussed in this study – such as CAR-T cell therapy and certain autologous cellular approaches – do not directly depend on unrelated donor registries, the anticipated persistence of allogeneic transplantation as a core therapeutic modality suggests that maintaining robust national donor registries will remain strategically important. In particular, donor registries are expected to retain substantial relevance for malignant and non-malignant indications in which allogeneic HCT remains clinically indicated despite the parallel expansion of non-registry-dependent advanced therapies.
Copyright (c) 2026 Danielli Oliveira, Bernardo Cabral, Roseli Monteiro, Carlos Conte Filho, Pamela Lopes, Inara Amorim, Daniel Silva, Islaine Santana, Marcela Bhering https://creativecommons.org/licenses/by/4.0
Challenges in the management of patients with chronic myelomonocytic leukemia in the Brazilian public healthcare system: from diagnosis to transplantation
Chronic myelomonocytic leukemia (CMML) represents a diagnostic and therapeutic challenge in the Brazilian public healthcare system, particularly due to limited access to advanced diagnostic tools and disease-modifying therapies. A retrospective chart review was conducted, including patients diagnosed with CMML from 2015 to 2025 at a university hospital in northeastern Brazil. Fourteen patients were identified, with a mean age of 58.7 years, and 50% were male. At diagnosis, 57.1% were in the chronic phase and 42.9% in the acute phase. Only two patients (14.3%) received hypomethylating agents, and four (28.6%) underwent allogeneic hematopoietic stem-cell transplantation. The median overall survival for the cohort was 25 months. Patients diagnosed in the acute phase had a shorter median overall survival compared to those diagnosed in the chronic phase (8 vs. 25 months). A trend toward improved survival was observed among transplanted patients (25 vs. 8 months). Compared with contemporary data, patients in this cohort had lower exposure to hypomethylating therapy, although transplant-related characteristics and early engraftment outcomes were similar. Structural and therapeutic limitations within the public healthcare system may impact disease management and outcomes, reinforcing the importance of improving access to diagnostic resources, disease-modifying therapies, and timely referral for transplantation.
Emanuelly Thays Muniz Figueiredo Silva Vasconcelos
Copyright (c) 2026 Emanuelly Thays Muniz Figueiredo Silva Vasconcelos, Felipe de Menezes Cunha, Gustavo Mesquita de Oliveira, Maria Laura Fontoura Chagas Rocha, Hércules Amorim Mota Segundo, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Hematopoietic cell transplantation outcomes in public centers in Brazil: analysis of the Brazilian Registry of Hematopoietic Cell Transplantation and Cellular Therapy, 2012–2024
This study provides a national overview of hematopoietic cell transplantation (HCT) outcomes in the Brazil’s public healthcare network, using data consolidated in the Brazilian Registry of Hematopoietic Cell Transplantation and Cellular Therapy (RBTCH-TC). A total of 5,923 first HCTs performed between 2012 and 2024 across 19 public centers were analyzed. Median follow-up among survivors reached 24.5 months, reflecting recent improvements in follow-up completeness and data reliability within the registry. Overall survival, relapse, and non-relapse mortality (NRM) rates were generally consistent with international benchmarks, despite the resource limitations characteristic of a publicly funded system. NRM was concentrated in the first year after allogeneic HCT, whereas relapse remained the predominant cause of late mortality across both autologous and allogeneic procedures. Acute graft versus-host disease (GVHD) was more frequent among recipients of mismatched related and unrelated donors, while chronic GVHD occurred more often in matched related donor transplants. These findings highlight the complexity of transplant care in the public setting and underscore the importance of standardized data collection, integration across centers, and long-term surveillance to support continuous quality improvement. Strengthening national collaborative initiatives such as the RBTCH-TC remains essential for guiding evidence-based strategies and improving patient outcomes throughout Brazil’s public transplant network.
Copyright (c) 2026 Anderson João Simione, Cinthya Corrêa da Silva, Nelson Hamerschlak, Afonso Celso Vigorito, Heliz Regina Alves das Neves, Adriana Seber, Carmem Maria Sales Bonfim Bonfim, Vergilio Antonio Rensi Colturato, Samir Kanaan Nabhan, Vanderson Geraldo Rocha, George Maurício Navarro Barros, Decio Lerner, Liane Esteves Daudt, Juliana Folloni Fernandes, Marcos Paulo Colella, Alexandre Silvério, Rodolfo Daniel de Almeida Soares, Gustavo Machado Teixeira, Victor Gottardello Zecchin, Abrahão Elias Hallack Neto, Rony Schaffel, Rodolfo Froes Calixto, Simone de Castro Resende Franco, Gianne Donato Costa Veloso, Vaneuza Araújo Moreira Funke, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Frailty score analysis of multiple myeloma patients in the evaluation prior to autologous hematopoietic stem cell transplantation
Introduction: Multiple myeloma (MM) is a common hematological neoplasm that is more prevalent at older ages. Autologous stem cell transplantation (ASCT) remains a relevant treatment option. The International Myeloma Working Group (IMWG) developed a score to assess frailty, dividing patients into fit, intermediate-fit, and frail. This study aims to analyze the profile, using the IMWG frailty score, of MM patients evaluated for ASCT. Methods: Retrospective, with a prospective arm, observational, descriptive study based on the review of records of MM patients evaluated for ASCT using the IMWG score in a Brazilian hospital from 2023 to 2025. Results: Eighty-one patients composed the sample, with a median age of 59 years. Using the IMWG frailty score, 57% of patients were classified as fit, 34% as intermediate-fit, and 9% as frail. Scoring on the IMWG score was decisive to contraindicating transplant (statistically significant difference compared to the fit group). There were no significant differences between groups concerning the toxicities, with a trend toward an increased risk of febrile neutropenia among frail patients (p = 0.058). The proportion of patients aged over 60 years was similar across groups. Conclusion: The IMWG frailty score is a valuable tool and reinforces the necessity of considering variables beyond age. Further discussion is required to validate these findings in diverse populations.
Beatriz Stela Gomes de Souza Pitombeira Araujo
Copyright (c) 2026 Felipe de Menezes Cunha, Hércules Amorim Mota Segundo, Livia Andrade Gurgel, Lucas Freire Castelo, Natalia Costa Bezerra Freire, João Paulo de Vasconcelos Leitão, Beatriz Stela Gomes de Souza Pitombeira Araujo, Karine Sampaio Nunes Barroso, Gustavo Mesquita de Oliveira, Maria Laura Fontoura Chagas Rocha, Emanuelly Thays Muniz Figueiredo Silva Vasconcelos, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Real-world outcomes of BCL2 inhibitor-based combination therapy in myelodysplastic syndrome and acute myeloid leukemia – Experience from a university hospital in northeastern Brazil
Acute myeloid leukemia (AML) and high-risk myelodysplastic neoplasms (MDS) predominantly affect elderly patients who are often ineligible for intensive chemotherapy. The emergence of BCL2 inhibitors, particularly venetoclax, has changed the therapeutic landscape in these populations when combined with hypomethylating agents or low-dose cytarabine. This study aims to describe the clinical and epidemiological characteristics, treatment patterns, and outcomes of patients with AML and high-risk MDS treated with venetoclax-based regimens at a university hospital in northeastern Brazil. The methodology consists of a retrospective, descriptive study including patients aged ≥ 18 years diagnosed from January 2020 to October 2025. Twenty-six patients were included (19 AML, seven MDS), with a median age of 65 years (range: 37-81). Among AML cases, 42.1% were de novo and 57.9% were secondary, mostly post-MDS. Overall, 46.1% achieved complete remission, and 3.8% achieved partial remission. The median time to best response was one cycle. Three patients (11.5%) underwent allogeneic hematopoietic stem cell transplantation. Hematologic toxicity was universal, with febrile neutropenia in 78.3%. Venetoclax-based regimens demonstrated response rates comparable to international real-world data in AML and high-risk MDS, even with shorter venetoclax duration. Frailty remains the main barrier to transplantation, highlighting the importance of expanding access to targeted therapies within the public health system.
Copyright (c) 2026 Gustavo Mesquita de Oliveira, Emanuelly Thays Muniz Figueiredo Silva Vasconcelos, Maria Laura Fontoura Chagas Rocha, Felipe de Menezes Cunha, Hercules Amorim Mota Segundo, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Acute liver failure as an initial presentation of multiple myeloma
Multiple myeloma (MM) is a clonal plasma cell neoplasm primarily involving the bone marrow and typically presenting with end-organ damage, including renal impairment, lytic bone lesions, anemia, and hypercalcemia. Extramedullary disease at diagnosis is uncommon, and acute liver failure as an initial manifestation is exceptionally rare; consequently, only a limited number of cases have been reported. We describe a 44-year-old woman with no relevant past medical history who was admitted to the intensive care unit with acute liver failure. After an extensive diagnostic work-up, MM with hepatic involvement was identified, and treatment was initiated promptly. Recognition of this unusual presentation is critical given its adverse prognosis, and reporting additional cases may help improve awareness and facilitate earlier diagnosis and management.
Copyright (c) 2026 Naddya Olivero Danies, Martin Milanesio, Ana Victoria Sanchez, Julieta Olmedo, Gaston Caeiro, Ana Lisa Basquiera https://creativecommons.org/licenses/by/4.0
Allogeneic hematopoietic stem cell transplantation in patients with sickle cell disease with alternative donors – Case series
Sickle cell disease is a hereditary hemoglobinopathy characterized by the presence of hemoglobin S (HbS), which leads to erythrocyte deformation, promotes hemolysis, and causes recurrent vaso-occlusive crises with multisystem involvement. Therapeutic options include pharmacological agents such as hydroxyurea, chronic blood transfusions, and, in selected cases, allogeneic hematopoietic stem cell transplantation (allo-HSCT), which remains the only curative option currently available. Here, we describe four cases that underwent allo-HSCT due to severe disease-related complications, highlighting the effectiveness of the procedure in appropriately selected cases.
Copyright (c) 2026 Marlene Micaela Sepich, Milagros Lopez Orozco, Gonzalo Ariel Ferini, Martin Milanesio, Maria Emilia Mas, Jorge Alberto Arbelbide, Ana Lisa Basquiera https://creativecommons.org/licenses/by/4.0
Clonal histiocytosis associated with evolving acute myeloid leukemia: a case report and literature review
Histiocytoses are rare disorders that may occur in association with hematologic malignancies, although reports preceding acute myeloid leukemia (AML), particularly in adults, remain scarce. We report the case of a 58-year old female patient who presented with persistent fever, weight loss, and progressive lethargy. Initial laboratory evaluation demonstrated pancytopenia, and bone marrow studies revealed erythroid and megakaryocytic dysplasia, plasmacytosis, and hemophagocytic activity, along with extensive histiocytic infiltration containing Langerhanslike giant cells. Immuno phenotyping at that time was negative for leukemia, and immuno histochemistry showed CD163 and S100 positivity, with no CD1a expression. The patient was initially managed with dexamethasone due to a suspected hemophagocytic lymphohistiocytosis (HLH)-like inflammatory syndrome, without adequate clinical response. Cytogenetic analysis revealed a clonal abnormality involving 11q23 in a subset of metaphases. After approximately 8 weeks, disease evolution was observed, and repeat bone marrow examination demonstrated 20% myeloblasts, with immunophenotypic findings consistent with AML. The patient, considered clinically pre-frail, received low-intensity chemotherapy with cytarabine in combination with venetoclax. Despite treatment, measurable residual disease persisted after two cycles, and she developed progressive clinical deterioration complicated by recurrent multidrug resistant infections, ultimately evolving to death. This case highlights the diagnostic complexity of early clonal myeloid neoplasms presenting with histiocytic and hemophagocytic features and emphasizes the importance of recognizing HLH as a possible secondary and unconfirmed inflammatory syndrome in the setting of evolving hematologic malignancy. Further studies are needed to clarify the biological mechanisms linking histiocytic proliferations and progression to myeloid neoplasms.
Copyright (c) 2026 Maria Laura Rocha, Emanuelly Thays Muniz Figueiredo Silva Vasconcelos, Gustavo Mesquita de Oliveira, Felipe de Menezes Cunha, Karla Hauanna Almeida Chaves do Vale, Francisco Dário Rocha Filho, Fernando Barroso Duarte https://creativecommons.org/licenses/by/4.0
Post-transplantation cyclophosphamide-based graft-versus-host disease prophylaxis in a patient transplanted from a matched related but non-sibling donor
While post-transplantation cyclophosphamide (PTCy)-based prophylaxis of graft-versus-host disease (GVHD) was increasingly used not only in haploidentical transplantation but also in transplantation from matched related donors, its benefit in the setting of matched non-sibling donors (MNSD) had not been discussed or reported. A 70-year-old Japanese male who was diagnosed with acute myeloid leukemia with internal-tandem duplication of the FLT3 gene was treated with intensive chemotherapy and quizartinib. After one cycle of consolidation, he was transplanted from an HLA 8/8 matched daughter. GVHD prophylaxis consisted of PTCy, tacrolimus, and mycophenolate mofetil. Neutrophil engraftment was attained at day 16, and he was still in remission at 1 year without complication, but except or grade II acute skin GVHD. Although there had been few reports on PTCy-based GVHD prophylaxis in transplantation from MNSD, our report suggested that these patients could benefit from this type of GVHD prophylaxis.
Copyright (c) 2026 Kazutoshi Ebisawa, Takahiro Takeuchi https://creativecommons.org/licenses/by/4.0
Gynecological chronic graft-versus-host disease: screening, diagnosis, and treatment. Brazilian Society of Cell Therapy and Bone Marrow Transplantation consensus
Objectives: To provide an updated review of the diagnosis, preventive measures, screening, and treatment of genital involvement in chronic graft-versus-host disease (cGVHD). Methods: A literature review was conducted using the PubMed and Google Scholar databases in January 2026. Results: The review addresses risk factors, clinical manifestations and classification, preventive strategies, and the treatment of both acute manifestations and late complications of genital cGVHD. Conclusion: Genital cGVHD is a common complication among women undergoing hematopoietic cell transplantation (HCT), with varying degrees of severity. We recommend gynecological follow-up for patients of all ages, beginning in the pre-transplant period, including educational and preventive measures as well as counseling on warning signs to enable early diagnosis and prompt treatment of genital cGVHD, thereby preventing long-term sequelae.
Copyright (c) 2026 Lenira Mauad, Andrea Maria Novaes Machado https://creativecommons.org/licenses/by/4.0
Autologous hematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis: A single-center institutional care protocol
Relapsing-remitting multiple sclerosis (RRMS) may remain clinically and radiologically active despite sequential exposure to high-efficacy disease-modifying therapies. In carefully selected patients with highly active inflammatory RRMS, autologous hematopoietic stem cell transplantation (AHSCT) has demonstrated superior inflammatory control and durable rates of no evidence of disease activity, with low treatment-related mortality rates in experienced centers. AHSCT promotes immune renewal through high-dose immunoablation followed by autologous stem cell rescue, aiming to restore immune tolerance. Here, we describe a structured institutional standard-of-care protocol for AHSCT in RRMS, developed to support safe referrals and harmonize neurology-hematology collaboration within a Brazilian tertiary center. By consolidating a transparent and safety-focused care pathway aligned with national and international guidance, this manuscript aims to facilitate standardized patient selection, optimize risk mitigation, and strengthen multidisciplinary communication between referring neurologists and transplant teams.
Copyright (c) 2026 Pedro D'elia Machado Silva, Stephanie Jauquin de Abreu, Alini Vargas, Maria Cecilia Aragon de Vecino https://creativecommons.org/licenses/by/4.0
Post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis and its impact on allogeneic hematopoietic cell transplantation donor selection: A summary of the SBTMO/ABHI joint session at the 2025 SBTMO annual meeting
This brief communication summarizes the joint session of the Brazilian Cellular Therapy and Bone Marrow Transplantation Society (SBTMO) and the Brazilian Histocompatibility and Immunogenetics Association (ABHI), held during the 2025 SBTMO Annual Meeting. The joint session focused on how post-transplant cyclophosphamide (PTCy) is redefining donor selection in contemporary allogeneic hematopoietic cell transplantation (HCT). In three lectures with experts in the field, several topics concerning PTCy were addressed, including the effectiveness of PTCy in overcoming the HLA barrier, the increased use of mismatched unrelated donors (MMUD) under PTCy, which significantly broadens HCT access for underserved ethnic minorities, and strategies for reducing the dose of PTCy. The impact of donor-specific HLA antibodies in allogeneic HCT, the importance of HLA serotypes (associated antigens) in improving the accuracy of virtual crossmatch during MMUD searches, and the utility of novel bioinformatics tools to enhance serotype evaluation for MMUD selection under PTCy were also discussed. Additionally, the present report outlines the discussions among the speakers, session chairs, and audience members.
Raquel Aparecida Salustriano Fabreti de Oliveira
Copyright (c) 2026 Alberto Cardoso Martins Lima, Antonio Martin Jimenez-Jimenez, Celso Arrais Rodrigues da Silva, Adriana Seber, Raquel Aparecida Salustriano Fabreti de Oliveira, Margareth Afonso Torres, Carmem Bonfim https://creativecommons.org/licenses/by/4.0