Mary Evelyn Dantas Flowers

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.

The Brazilian Hematopoietic Cell Transplantation and Cellular Therapy (HCT-CT/BR) Registry depends on data manager (DM) training to ensure sustainability, for which it is essential to standardize processes. The objectives of this study were to describe the development of a support manual for Brazilian DMs reporting to the Center for International Blood and Marrow Transplant Research and to present quality assessment results. The descriptive study (Feb–Aug 2024) included structuring, content creation, and dissemination of the manual. Survey assessed satisfaction. The manual was launched at the XXVIII Brazilian Society of Cellular Therapy and Bone Marrow Transplantation Congress. Thirty-nine professionals rated it 9.3/10 for satisfaction and 9.4/10 for recommendation. A manual is crucial for standardizing processes and ensuring data quality, offering clear and up-to-date guidance.

Systematic evaluation of hematopoietic cell transplantation (HCT) outcomes is essential to improve clinical practice and meet international quality standards. In Brazil, the partnership between the Brazilian Society of Cellular Therapy and Bone Marrow Transplantation (SBTMO) and the Center for International Blood and Marrow Transplant Research (CIBMTR) has advanced national data reporting and benchmarking through the Hematopoietic Cell Transplantation Brazilian Registry (HCTBR). This study reports outcomes from 11,210 first HCTs (5,984 allogeneic; 5,226 autologous) performed between 2012 and 2023 across 44 Brazilian centers. Median recipient age was 29 years for allogeneic and 53 years for autologous transplants. Acute leukemias predominated among allogeneic cases, while multiple myeloma was the most common indication for autologous HCT. Two-year overall survival was 82.0% for autologous and 59.8% for allogeneic transplants, with variation by donor type (matched related 61.8%, mismatched related 54.0%, unrelated 63.3%). Two-year non-relapse mortality was 8.0% and 21.6% for autologous and allogeneic transplants, respectively. The cumulative incidence of grade II–IV acute graft-versus-host disease (GVHD) at two years was 29.9%, with chronic GVHD incidence of 29.5%. Two-year relapse incidence was 24.1% for allogeneic and 25.8% for autologous HCT. Despite challenges within the Brazilian healthcare system, these outcomes align with international registry data. Adequate data completeness supports the robustness of these findings. Our results highlight the quality of Brazilian transplant programs and underscore the value of standardized outcome monitoring to foster continuous improvement. Strengthening center participation, follow-up, and data management remains critical to maintaining registry quality and enhancing patient care.

The development of the Brazilian Registry of Hematopoietic Cell Transplantation (HCT) in collaboration with the Center for International Blood and Marrow Transplant Research (CIBMTR) continues to provide a comprehensive assessment of the activity and general outcomes of HCT in Brazil. In this paper, we report an update of such activity. Brazilian transplant centers report their data to the CIBMTR, using the FormsNet3 platform. The data then return to the Brazilian Cellular Therapy and Bone Marrow Transplant Society (SBTMO) through the Data Back to Centers (DBtC) tool. Data from patients who received an HCT from 2012 to 2024 from Brazilian centers were extracted from the CIBMTR. A descriptive analysis was carried out using patient, disease and transplant-specific variables and overall survival analysis using the Kaplan Meier method. A total of 14,331 patients were eligible for this study (6,583 autologous and 7,748 allogeneic HCTs). The number of reporting centers increased from 40 to 45 during the period. The most common HCT indication in Brazil for allogeneic HCT was AML, with 171 HCTs per year and, for autologous HCT, multiple myeloma, with 273 HCTs per year. Among allogeneic HCT, in the last 4 years, mismatched related donor was the main source of donors. Regarding the graft source for allogeneic HCTs, bone marrow (BM) was the most frequent among pediatric HCTs, while peripheral blood (PB) was the most used in adults. Infections were the leading cause of death in the first 100 days after all types of HCTs. Patients with acute leukemia who underwent HCT at an advanced disease stage had lower survival rates compared to those at earlier stages. Despite the differences in the number of cases and follow-up time, the results in this study were similar to those presented in the United States (USA) Summary Slides.

After allogeneic hematopoietic cell transplantation (HCT), a very small number of donor stem cells reconstitute the recipient hematopoietic system, whereas the donor is left with a near-normal pool of stem cells. We hypothesized that the increased replicative stress on transplanted donor cells in the recipient could lead to the disproportionate proliferation of clonal hematopoiesis (CH) variants. We obtained blood samples from 16 related donor-recipient pairs at a median of 33.8 years (range: 6.6 to 45.7) after HCT, including the longest surviving HCT recipients in the world. For 11 of 16 pairs, a donor sample from the time of HCT was available for comparison. We performed ultrasensitive duplex sequencing of genes recurrently mutated in myeloid malignancies and CH, as well as a set of functionally neutral genomic regions representative of human genomic content at large. CH variants were observed in all donors, even those as young as 12 years old. Where donor pre-HCT sample was available, the average mutation rate in donors compared to recipients post-HCT was similar (2.0% versus 2.6% per year, respectively) within genes recurrently mutated in myeloid malignancies. Twenty-two (5.6%) of the 393 variants shared between paired donors and recipients post-HCT showed ≥10-fold higher variant allele frequency (VAF) in the recipient. A longer time since HCT was positively associated with the expansion of shared variant VAFs in the recipient. In conclusion, even decades after HCT, there does not appear to be widespread accelerated clonal expansion in the transplanted cells, highlighting the immense regenerative capacity of the human hematopoietic system.

No período de agosto/86 a fevereiro/87, três pacientes foram tratados com VP-16-1800mg/m2, em infusão única em 26 horas no dia -6, seguida por Ciclo fosfamida (EDX) 50mg/kg/d nos dias -4 e -3. Irradiação corpórea total (ICT) 1000 cGy em 5 frações de 200cG y nos dias -2, -1 e 0. O transplante de medula óssea (TMO) foi realizado no dia O utilizando doadores HLA compatíveis. O estudo incluiu três pacientes: um paciente portador de Leucemia Linfoblástica Aguda (LLA) em 1.ª recaída medular; um paciente portador de Leucemia Mielóide Crônica (LMC) em 2.ª fase crônica e um paciente portador de LLA em 2.ª remissão. A idade variou entre quatro e 29 anos. A principal toxicidade apresentada por este regime foi mucosite severa que ocorreu nos três pacientes. Todos os três pacientes apresentaram ulcerações orais e um deles apresentou diarréia hemorrágica com volume superiora dois litros. Em todos os pacientes o quadro se resolveu nas três semanas que se seguiram ao TMO. Um paciente apresentou alterações eletrocardiográficas significativas sem, entretanto, ocorrerem manifestações clínicas de cardiotoxicidade. A eficácia do regime pode ser testada no paciente em recaída que permanece em remissão 210 dias pós-TMO. No paciente portador de LMC houve eliminação do cromossomo Ph1. Concluímos que VP-16/EDX/ICT é um regime eficaz e bem tolerado no tratamento de neoplasias hematológicas.