ABSTRACT Introduction: Brazil is one of the countries with the largest population of people with hemophilia (PwH) worldwide. In this scoping review, we aim to investigate the Brazilian context for hemophilia regarding three predefined concepts: (i) clinical-epidemiological profile, (ii) burden of disease and (iii) patient journey and unmet needs. Methods: Three questions in each concept guided the screening of references retrieved by systematic searches carried out in MEDLINE, LILACS and the Digital Library of Theses and Dissertations. Quantitative and qualitative studies conducted in Brazil from 2002 onwards were assessed for eligibility. Main results: Ninety-two studies were included. A total of 66 studies addressed the concept “Clinical-epidemiological profile”, 31 investigated the concept of “Burden of disease” and 26 addressed the concept “Patient journey and unmet needs”. Based on these studies, pain and arthropathy affect a substantial proportion of the PwH, with physical functioning, pain and school or work being the domains of quality of life with the greatest impact. About 43 % to 82.6 % of the PwH are unemployed. Rates of inhibitor development are highly variable across studies, especially in hemophilia A. Adherence to prophylactic treatment ranges from 25 % to 72 %. The annualized bleeding rate is estimated at 2.4 ± 4.1. The barriers to treatment identified include distance to reference centers, lack of coordination of specialized and emergency care and restricted access to rehabilitation. Conclusions: Hemophilia poses a considerable burden on the PwH. Despite the available modalities of treatment, there are remaining unmet needs that should be addressed by researchers and policy makers in the future. Introduction (PwH worldwide review concepts i (i clinicalepidemiological clinical epidemiological profile ii (ii iii (iii Methods MEDLINE Dissertations 200 eligibility results Ninetytwo Ninety two included 6 Clinicalepidemiological Clinical , profile” 3 Burden 2 Patient . needs” functioning impact 4 826 82 82. unemployed 7 24 2. 41 1 4.1 centers rehabilitation Conclusions future 20 8 4.

ABSTRACT Background: Immunosuppressive therapy is the standard management of adults with aplastic anaemia. Antithymocyte globulin is used as first-line treatment of patients not eligible for bone marrow transplantation. This being a rare disease, available evidence in India is scarce. This study aimed to present experience in treating adult aplastic anaemia patients by immunosuppressive therapy using antithymocyte globulin-equine (Thymogam) in two tertiary care centres of northeast India. Methods: This case series was conducted at the Health city hospital, Guwahati, and Excel Care Hospital, Guwahati from 2018 to 2020. Eighteen adult aplastic anaemia patients who were treated by immunosuppressive therapy with antithymocyte globulin-equine (Thymogam) and followed up for two years were included. Treatment response and relapse are described. Results: All the 18 patients, (14 severe, four very severe) were uniformly treated with immunosuppressive therapy (Thymogam 40 mg/kg/d for four days with oral Cyclosporine from Day-1). Cyclosporin A was used as a concomitant drug in 94.44 % of the patients. At two years of follow up, 66.7 % showed a response and the mortality rate was 11.1 %. Conclusion: The results of this case series substantiate the effectiveness of immunosuppressive therapy with a low-cost preparation of horse antithymocyte globulin (Thymogam) along with cyclosporin A in the management of aplastic anaemia patients not suitable for bone marrow transplantation. Background firstline first line transplantation disease scarce globulinequine equine Thymogam Methods hospital Hospital 201 2020 included described Results 1 14 (1 severe 4 mgkgd mg kg d Day1. Day1 Day . Day-1) 9444 94 44 94.4 667 66 7 66. 111 11 11. Conclusion lowcost low cost 20 202 ( Day-1 944 9 94. 6 2 Day-

ABSTRACT Introduction: Beta-thalassemia major patients need a regular blood transfusion to have an initial normal growth. However, these patients have an increased risk of developing alloantibodies. Our main goal was to study HLA alloimmunization in Moroccan Beta-thalassemia patients by confronting it with transfusion and demographic criteria, exploring the involvement of HLA typing profile in the development of HLA antibodies and in turn determining risk factors for their development. Methods: The study consisted of 53 Moroccan pediatric patients with Beta-thalassemia major. Screening for HLA alloantibodies was performed using Luminex technology Whereas HLA genotyping was done with sequence-specific primers (PCR-SSP). Results: In this study, 50.9% of patients have been identified as positive for HLA antibodies, with 59.3% having both HLA Class I and Class II antibodies. A significant increase frequency of DRB1*11 allele was revealed in non-immunized patients (34.6% vs. 0%, p = 0.001). Our results also revealed that the majority of our HLA immunized patients were women (72.4% vs. 27.6%, p = 0.001), and transfused with more than 300 units of RBC units (66.7% vs. 33.3%, p = 0.02). There were statistically significant differences when comparing these frequencies. Conclusions: This paper revealed that the transfusion dependent Beta-thalassemia major patients are exposed to risk of developing HLA antibodies following transfusions with leukoreduced RBC units. The HLA DRB1*11 was a protective factor against HLA alloimmunization in our beta-thalassemia major patients. Introduction Betathalassemia Beta thalassemia growth However criteria Methods 5 sequencespecific sequence specific PCRSSP. PCRSSP PCR SSP . (PCR-SSP) Results 509 50 9 50.9 593 59 3 59.3 DRB111 DRB DRB1 11 DRB1*1 nonimmunized non 34.6% 346 34 6 (34.6 vs 0 0% 0.001. 0001 0.001 001 0.001) 72.4% 724 72 4 (72.4 276 27 27.6% 0.001, , 30 66.7% 667 66 7 (66.7 333 33 33.3% 0.02. 002 0.02 02 0.02) frequencies Conclusions betathalassemia beta (PCR-SSP 50. 59. DRB11 1 DRB1* 34.6 (34. 000 0.00 00 72.4 (72. 2 27.6 66.7 (66. 33.3 0.0 34. (34 72. (72 27. 66. (66 33. 0. (3 (7 (6 (

ABSTRACT Introduction: High-dose cytarabine is considered standard of care as consolidation chemotherapy in adults with acute myeloid leukemia (AML) who are not eligible for allogeneic hematopoietic cell transplantation, but may be associated with significant toxicity. We evaluated the toxicity associated with high-dose cytarabine given as consolidation in AML patients treated at a Brazilian public hospital. Methods: We retrospectively reviewed the charts of all patients with AML treated between 2008 and 2020 who obtained complete remission (CR) after one cycle of induction chemotherapy and received consolidation with at least one cycle of high-dose cytarabine (defined as 3 g/m² every 12 h days 1, 3 and 5). Results: Among 61 patients who received induction remission, 32 obtained CR and 28 received at least one cycle of high-dose cytarabine, for a total of 67 cycles (median 2 cycles per patient, range 1 - 4). In 45 cycles (67.2%) the patient was discharged after the end of chemotherapy, with a median of 6 days at home (range 3 - 8). Readmission occurred in 31 of the 45 cycles (68.9%). The most frequent toxicities were febrile neutropenia (56.7%), nausea and vomiting (23.9%), oral mucositis (14.9%) and diarrhea (11.9%). Bacteremia was documented in 13 cycles (34.2%). There were three cases of typhlitis and two of invasive fungal disease (aspergillosis and candidemia). Four patients died (14.3%), with two deaths considered treatment-related (candidemia and typhlitis). Conclusion: In the setting of a Brazilian public hospital, high-dose cytarabine as consolidation therapy is feasible, with manageable toxicity profile. Introduction Highdose High dose (AML transplantation highdose high hospital Methods 200 202 (CR defined gm² gm g m² m g/m 5. 5 . 5) Results 4. 4 4) 67.2% 672 (67.2% 8. 8 8) 68.9%. 689 68.9% 68 9 (68.9%) 56.7%, 567 56.7% , 56 7 (56.7%) 23.9%, 239 23.9% 23 (23.9%) 14.9% 149 14 (14.9% 11.9%. 119 11.9% 11 (11.9%) 34.2%. 342 34.2% 34 (34.2%) aspergillosis candidemia. candidemia candidemia) 14.3%, 143 14.3% (14.3%) treatmentrelated treatment related typhlitis. typhlitis) Conclusion feasible profile 20 67.2 (67.2 68.9 (68.9% 56.7 (56.7% 23.9 (23.9% 14.9 (14.9 11.9 (11.9% 34.2 (34.2% 14.3 (14.3% 67. (67. 68. (68.9 56. (56.7 23. (23.9 14. (14. 11. (11.9 34. (34.2 (14.3 (67 (68. (56. (23. (14 (11. (34. (6 (68 (56 (23 (1 (11 (34 ( (5 (2 (3

ABSTRACT Objective: The aim of this study was to determine the association between dental tissues and sickle cell anemia (SCA) and how it impacts the quality of life related to oral health. Materials and Methods: It was a cohort study of 154 Congolese participants with and without SCA conducted in the dental service of SCA at the Yolo Center, Kinshasa, aged at least 6 years and without a history of clinically severe conditions (hospitalization and blood transfusion), who were regularly monitored. The inclusion criteria were the diagnosis confirmation of SCA at the health service in a period of at least 6 months before enrollment in this study. Dental tissues were assessed by a clinical examination using a dental mirror and probe. The index of Decayed-Missing-Filled Teeth (DMFT) was used to assess the dental state of the participants. For Oral Health-related Quality of Life (OHrQoL), the Congolese versions of the perception questionnaires, modified from the Oral Health Impacts Profile (OHIP-23), were used for participants. Each question had to be answered by yes or no, depending on whether the participant was satisfied (outcome = 1) or dissatisfied (outcome = 0) about an oral health-related quality of life. Results: Of the 154 participants, aged from 6 to 64 years, with a mean age of 19.5 ± 7 (SD) years, 96 presented with SCA and only 68 were correctly followed; 102 did not present SCA and only 86 were correctly followed. The DMFT and dmft indexes were higher in the SCA group, being 2.9 and 2.5, respectively. The difference between the SCA group and the control group was significant for decayed teeth, missing teeth, filled teeth and no caries. Of the different dimensions of quality of life that were compared between the SCA group and control group, 15 of 23 items were statistically significant. Conclusion: The present study strongly confirmed an association between dental caries and missing teeth with sickle cell anemia. Secondly, the quality of life for SCA participants seems to be poor, compared to the control group. Objective (SCA Methods Center Kinshasa hospitalization transfusion, transfusion , transfusion) monitored probe DecayedMissingFilled Decayed Missing Filled (DMFT Healthrelated OHrQoL, OHrQoL (OHrQoL) questionnaires OHIP23, OHIP23 OHIP (OHIP-23) outcome 1 0 healthrelated Results 195 19 5 19. SD (SD 9 followed 10 8 29 2 2. 25 2.5 respectively Conclusion Secondly poor (OHrQoL OHIP2 (OHIP-23 (OHIP-2 (OHIP- (OHIP

ABSTRACT Introduction: Dengue is the most fatal virus disease spread by mosquito bites and Aedes aegypti is the main transmitting agent. It is an endemic disease in the tropical and subtropical regions, currently affecting more than 100 countries. Although most patients present mild forms of the disease, a considerable proportion of individuals has severe alterations in the blood count. The aim of this study was to evaluate the consumption pattern of blood components in epidemic and non-epidemic periods and to verify if there was an impact on dengue cases and the death rate. Method: This is a retrospective cross-sectional study conducted through the collection and analysis of data from the Brazilian Ministry of Health from 2008 to 2019 on new cases and deaths from dengue, as well as the consumption of blood components in the period mentioned by hemovigilance bulletins of the Brazilian authority. Results: Regarding the results, no significant difference was found between the absolute amount of blood components used in years with an epidemic peak. Regarding the relative values, an important variation was shown among the distributive consumption patterns of blood components in the outbreak years. In the univariate linear regression analysis, there was statistical significance between the increase in the number of dengue cases and deaths from dengue with the increase in the consumption of red blood cell concentrates (RBP), platelet concentrates (PP), fresh frozen plasma (FFP) and cryoprecipitate (Cryo) (p-value < 0.05). The increase in dengue cases was related to the increase in Cryo consumption with clinical significance (R² > 0.5), but dengue deaths were not correlated to the same. In multivariate analysis, all regression models had clinical and statistical significance. Conclusion: The data obtained in the present study demonstrate that there is a relevant relationship between the increase in cases and deaths from dengue with the blood components usage, especially PP, FFP and cryoprecipitate. Introduction agent regions 10 countries count nonepidemic non rate Method crosssectional cross sectional 200 201 authority Results results peak values RBP, RBP , (RBP) PP (PP) (FFP (Cryo pvalue p value 0.05. 005 0.05 . 0 05 0.05) R² R (R 0.5, 0.5 5 0.5) same Conclusion usage 1 20 (RBP (PP 00 0.0 0. 2

ABSTRACT Acute megakaryoblastic leukemia is characterized by heterogeneous biology and clinical behavior. Immunophenotypic characteristics include the expression of megakaryocytic differentiation markers (e.g. CD41, CD42a, CD42b, CD61) associated with immaturity markers (CD34, CD117, HLA-DR) and myeloid markers (e.g. CD13, CD33) and even with lymphoid cross-lineage markers (e.g. CD7, CD56). Although the diagnostic immunophenotype has already been well described, given the rarity of the disease, its immunophenotypic heterogeneity and post-therapeutic instability, there is no consensus on the combination of monoclonal markers to detect minimal/measurable residual disease (MRD). Currently, MRD is an important tool for assessing treatment efficacy and prognostic risk. In this study, we evaluated the immunophenotypic profile of MRD in a retrospective cohort of patients diagnosed with acute megakaryoblastic leukemia, to identify which markers, positive or negative, were more stable after treatment and which could be useful for MRD evaluation. The expression profile of each marker was evaluated in sequential MRD samples. In conclusion, the markers evaluated in this study can be combined in an MRD immunophenotypic panel to investigate for megakaryoblastic leukemia. Although this study is retrospective and some data are missing, the information obtained may contribute to prospective studies to validate more specific strategies in the detection of MRD in acute megakaryoblastic leukemia. behavior e.g. eg e g (e.g CD41 CD CD42a CDa CD42b CDb b CD61 CD34, CD34 (CD34 CD117 HLADR HLA DR HLA-DR CD13 CD33 crosslineage cross lineage CD7 CD56. CD56 . CD56) described posttherapeutic post therapeutic instability minimalmeasurable minimal measurable MRD. (MRD) Currently risk negative evaluation samples conclusion missing e.g CD4 CD6 CD3 (CD3 CD11 CD1 CD5 (MRD (CD