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Hematopoietic cells transplants are technically complex and expensive imposing a huge burden on health care systems, especially those in developing countries and regions. In 2017 > 4500 transplants were done in 13 Latin American countries with established transplant programmes. We interrogated data on transplant rate, cost, funding source, hospital type, Gini coefficient and the United Nations Development Programme Inequality-Adjusted Human Development Index to determine co-variates associated with transplant development. Transplant rates varied almost 30-fold between the 13 countries from 345 in Uruguay to 12 in Venezuela with a regional transplant rate 7–8-fold lower compared with the US and EU. We found significant correlations between higher transplant cost, public funding, transplants in private hospitals with transplant rate. Low cost pertransplant regardless of payor and transplants done in public hospitals were associated with low transplant rates. In contrast, high cost pertransplant funded by the government and transplants done in private hospitals were associated with high transplant rates. Surprisingly, we found transplant rates were higher when transplants cost more, when they were done in private for-profithospitals and payed for with public funds. These data give insights how to increase transplant rates in Latin America and other developing regions.

Social determinants of health, including poverty, contribute significantly to health outcomes in the United States; however, their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research database. We assembled 2 pediatric cohorts undergoing first allogeneic HCT from 2006 to 2015 at age ≤18 years, including 2053 children with malignant disease and 1696 children with nonmalignant disease. Neighborhood poverty exposure was defined a priori per the US Census definition as living in a high-poverty ZIP code (≥20% of persons below 100% federal poverty level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS), defined as the time from HCT until death resulting from any cause. Secondary outcomes included relapse and transplantation-related mortality (TRM) in malignant disease, acute and chronic graft-versus-host disease, and infection in the first 100 days post-HCT. Among children undergoing transplantation for nonmalignant disease, neighborhood poverty was not associated with any HCT outcome. Among children undergoing transplantation for malignant disease, neighborhood poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplantation outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared with those with private insurance. These data suggest opportunities for future investigation of the effects of household-level poverty exposure on HCT outcomes in pediatric malignant disease to inform care delivery interventions.

Social determinants of health, including poverty, contribute significantly to health outcomes in the United States; however, their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research database. We assembled 2 pediatric cohorts undergoing first allogeneic HCT from 2006 to 2015 at age ≤18 years, including 2053 children with malignant disease and 1696 children with nonmalignant disease. Neighborhood poverty exposure was defined a priori per the US Census definition as living in a high-poverty ZIP code (≥20% of persons below 100% federal poverty level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS), defined as the time from HCT until death resulting from any cause. Secondary outcomes included relapse and transplantation-related mortality (TRM) in malignant disease, acute and chronic graft-versus-host disease, and infection in the first 100 days post-HCT. Among children undergoing transplantation for nonmalignant disease, neighborhood poverty was not associated with any HCT outcome. Among children undergoing transplantation for malignant disease, neighborhood poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplantation outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared with those with private insurance. These data suggest opportunities for future investigation of the effects of household-level poverty exposure on HCT outcomes in pediatric malignant disease to inform care delivery interventions.

Background: Diffuse Large B-Cell Lymphoma (DLBCL) is the most common B-cell non-Hodgkin lymphoma. DLBCL commonly affects patients with comorbidities and the elderly. To date, chemoimmunotherapy remains the standard of care for these patients. Data on patients aged ≥65 years managed in Latin America (LATAM) are scarce. Herein, we examine the clinical, treatment and outcome patterns in older patients with DLBCL, with a focus on the effect of treatment in survival outcomes.

Introduction:venetoclax plus azacitidine (Ven/Aza) is the standard-of-care in patients with newly diagnosed acute myeloid leukemia (AML) who are unsuitable candidates for intensive chemotherapy (IC), prompting questions related to whether it should be used for candidates fit for IC (FIT). Access to novel agents in low- and middle-income countries (LMIC) is limited, however, in a previous phase 2 study (NCT05048615), we demonstrated that using venetoclax concomitantly with strong CYP3A4 inhibitors like itraconazole could be a practical approach to reduce the venetoclax dose and diminish treatment-related costs without compromising treatment response. We sought to compare FIT patients for IC with newly diagnosed AML who received either low-dose Ven/Aza or traditional IC as first-line treatment to evaluate survival outcomes in LMIC.

INTRODUCTION:Methotrexate is a fundamental drug in the treatment of acute lymphoblastic leukemia (ALL), playing an important role in CNS prophylaxis. Two strategies of methotrexate intensification have been studied: high-dose regimens (with leucovorin salvage) and Capizzi regimen (escalating doses, without salvage, with addition of L-asparaginase). Fifty-five percent of pediatric patients with ALL in Mexico are treated in general hospitals, with limited hospital bed availability, which may represent a deferral of treatment. We wanted to explore a feasible, economical and accessible ambulatory scheme, which may represent an option for those centers that do not have the infrastructure for maintenance with HDMTX.

Background

Background:BCR::ABL1 tyrosine kinase inhibitors (TKIs) in combination with chemotherapy or steroids remain the standard of care in patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Most patients treated with first- or second-generation TKIs eventually experience disease progression due to treatment resistance. Ponatinib is the only currently approved pan-BCR::ABL1 inhibitory TKI that potently inhibits wild-type and single resistance mutation variants of BCR::ABL1, including T315I. Multiple studies have shown promising minimal residual disease (MRD) negativity (neg) rates and survival outcomes with ponatinib in combination with chemotherapy or chemotherapy-free regimens. PhALLCON (NCT03589326) is the first randomized study comparing frontline TKIs in adults with Ph+ ALL. The primary endpoint for PhALLCON was met, with a clinically significantly higher MRD-neg complete remission (CR) rate at end of induction (EOI) with ponatinib vs imatinib (34.4% vs 16.7%; risk difference: 0.18 [95% confidence interval (CI): 0.06‒0.29]; P=0.0021) and a manageable safety profile comparable with imatinib. Here we report subgroup efficacy analyses from PhALLCON.

Introduction:Multiple sclerosis (MS) is a disabling disease that affects young adults. Treatments for MS have increased exponentially in number, efficacy and risk. Autologous hematopoietic stem cell transplantation (aHSCT) can change the natural history of the disease. To analyze if aHSCT should be done early in the course of the disease or after failing of other therapies, we have studied the long-term results of aHSCT in a cohort of persons with MS

Introduction:Up to one-third of patients with Hodgkin's lymphoma (HL) are not responsive to first-line therapy or eventually relapse. For these patients, second-line chemotherapy followed by autologous stem cell transplantation (ASCT) represents the standard of care, with better results for those that respond to salvage chemotherapy, usually achieving higher survival after ASCT, however, this strategy is far from perfection.

Introduction: Acute leukemias represent an important public health problem due to their high morbidity and mortality. Treatment failure due to abandonment or loss of follow-up (LOF) is a contributing factor to poor outcomes.

Background

In low- and middle-income countries (LMIC) like Mexico, the treatment of patients with refractory or relapsed acute lymphoblastic leukemia (r/r ALL) represents a significant challenge due to limited access to immunotherapy and hematopoietic stem cell transplantation (HSCT). As a result, the one-year survival rate is reported to be less than 20%. The use of intensive chemotherapy has not improved survival in this group due to its high toxicity.

IntroductionThe standard treatment for newly diagnosed (ND)-CML is tyrosine kinase inhibitors (TKIs) such as dasatinib. However, the cost of treatment with these drugs can be prohibitively high. The CYP inhibition strategy involves intentionally inhibiting a specific enzyme, in this case CYP3A4, to increase plasma concentration of dasatinib and reduce the required dose from 100mg to 25mg daily.

Introduction:Diffuse Large B-cell lymphoma (DLBCL) is an aggressive malignancy and the most common type of non-hodgkin´s lymphoma. Biologically, survival of neoplastic lymphoid cells depends on self-mediated mechanisms and the tumor microenvironment ( Steen CB,2021) leading to an inflammatory state. Although a better understanding of lymphoma biology has helped predict outcomes in DLBCL, modern tools such as next generation sequencing are not available in Latin America ( LATAM). The systemic immune-inflammation index (SII) is a score based on neutrophil, platelet, and lymphocyte counts that has proven to be useful in DLBCL ( ZanZan W, 2021 & Xiao-Bo,2021). Moreover, its variables can be easily evaluated at resource-limited healthcare setting. Thus, we aim to validate this new SII score in our LATAM DLBCL population.

Introduction. Diffuse large B-cell lymphoma ( DLBCL) is the most common type of malignant lymphoid neoplasm. The international Prognostic Index (IPI) and its variants are the main prognostic tools used in DLBCL with value in the rituximab era. Certain molecular biomarkers and genetic signatures in DLBCL have been identified, but the cost and unavailability in Latin America ( LATAM) are an issue. Therefore, using accesible tools in LATAM is a potencially unmet need. Vlatka (2021), reported a new score (HALP: hemoglobin, albumin, lymphocytes, and platelets) for overall survival (OS) in DLBCL. These parameters are easy to access in LATAM centers. Thus, we aim to validate this novel OS score in the GELL database, and expanding database from LATAM countries.

Introduction: the practice of hematology is in continued transformation fueled by scientific advances. However, access to training and education is not equally distributed worldwide, especially for under-represented groups such as trainees living in low and middle-income countries (LMICs). Differences in education and funding in the field remain understudied. Our goal was to identify and review the different educational and funding opportunities offered by national and international hematology-focused academic societies. We aimed to provide an overview of the current state of continuing education in hematology and present gaps and areas of opportunity.

Introduction

Villela: amgen: Speakers Bureau; Roche: Other: advisory board, Speakers Bureau. Idrobo:Janssen: Honoraria, Speakers Bureau; Amgen: Honoraria, Speakers Bureau; Abbvie: Honoraria, Speakers Bureau; Tecnofarma: Honoraria, Speakers Bureau; Takeda: Honoraria, Speakers Bureau. Gomez-Almaguer:Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AbbVie: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene/BMS: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Castillo:Janssen: Consultancy, Research Funding; TG Therapeutics: Research Funding; Kymera: Consultancy; Abbvie: Research Funding; Beigene: Consultancy, Research Funding; Pharmacyclics: Consultancy, Research Funding.

Introduction. There are different scoring systems to differentiate risk groups in patients with DLBCL treated with chemoimmunotherapy. Those systems have used the same 5 variables (age, performance status, LDH, stage, extranodal involvement) for 27 years. However, LATAM data have not been included in the development of previous scoring systems. It is important to mention that novel biological variables, such as albumin, beta-2-microglobulin (B2M) and platelet/lymphocyte ratio (PLR), have been reported and could improve discrimination (Villela et al. Blood 2019; 134Suppl_1: 1613). Therefore, we carried out a large, multinational study to develop and validate a LATAM-IPI score.

Background: The COVID pandemic has resulted in significant changes many aspects of daily living. To understand the impact that COVID-19 has had on the myeloproliferative neoplasm (MPN) patient population, we conducted an internet based survey.

We studied rates of granulocyte and platelets recovery in 359 consecutive subjects receiving blood cell infusions in the context of autotransplants for plasma cell myeloma (N= 216) and lymphomas (N= 143). Blood cells were mobilised with filgrastim given for 4–5 days and collected after a median of 2 (range, 1–2) apheresis. Apheresis products were stored at 4° C for a median of 3 days (range, 2–6 days). Most subjects received carmustine, etoposide, cytarabine and melphalan (BEAM), cyclophosphamide, carmustine and etoposide (CBV) or high-dose melphalan. Filgrastim was given post transplant to 319 subjects. Median numbers of mononuclear cells collected was 31 × 10E + 6/kg (interquartile range (IQR) 37 × 10E + 6 cells/kg). Median numbers of CD34-positive cells collected was 3.6 × 10E + 6/kg (IQR 3.8 × 10E + 6/Kg). Median viability after collection was 90% (IQR 7%) after storage, 88% (IQR 12%). A total of 255 of 256 evaluable subjects recovered bone marrow function and there was no late bone marrow failure. Median interval to neutrophils >0.5 × E + 9/L was 13 days (range, 9–39 days) and to platelets >20 × 10E + 9/L, 16 days (range, 7–83 days). These rates and ranges seem comparable to those reported after autotransplants of frozen blood cells. There was no correlation between numbers of storage days at 4 °C and viability afte storage (r= −0.018, p= 0.14)) nor rates of recovery of neutrophils (r= −0.054, p= 0.52) or platelets (r= 0.116, p= 0.14). Blood cells collected for autotransplant can be stored at 4 °C for 6 d. This method is simple, inexpensive and widely applicable.

Gomez-Almaguer: Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AbbVie: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene/BMS: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Roche: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.

Gomez-Almaguer: AstraZeneca:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Roche:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Pfizer:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Celgene/BMS:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Amgen:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Novartis:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;AbbVie:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau;Janssen:Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.

Introduction

Introduction

Aplastic anaemia (AA) consists of pancytopenia and empty bone marrow. Its incidence varies worldwide but predominates in developing countries. Diverse aetiologies are involved, with autoimmunity at the centre of the p-icture. For the 70% of patients with the severe and very severe forms of AA and who lack a human leukocyte antigen (HLA)-matched sibling, immunosuppressive therapy (IST) is key in treating the disease, with a remission rate close to 70%, an 80 – 90% 5-year survival rate in responding patients and a relapse rate close to 10%. For the 30% with a sibling donor available, h-aematopoietic stem cell transplant (HSCT) from bone marrow or peripheral blood has up to a 90% chance of cure, with a 5 – 10% graft rejection/failure rate. Patients who fail IST (25 – 30%) and lack a sibling donor can benefit from CD34+-enriched, p-artially T cell-depleted unrelated stem cell transplants, with a g-eneral survival rate up to 37%, the newest source of stem cells for this modality being cord blood. Non-myeloablative, irradiation-free co-nditioning regimens offer appreciable benefits, and new immunosuppressive agents, such as fludarabine and alemtuzumab, have been incorporated with promising preliminary results. Graft-versus-host disease, graft failure and infections remain significant challenges in HSCT for which innovative treatment strategies are being developed at present.

Background:Up to one-third of patients with Hodgkin lymphoma (HL) are not responsive to first-line therapy or eventually relapse. New drugs, including anti-PDL1 (i.e., nivolumab) and monoclonal antibodies (i.e., brentuximab vedotin (BV)), have changed the landscape in the treatment of HL. However, the financial toxicity associated with these new drugs is worrisome and are practically inaccessible to most patients living in low-middle income countries. The approved dose of nivolumab in refractory classical Hodgkin Lymphoma (HL) is 3 mg/kg, but lower doses have demonstrated efficacy.

Introduction

Background:Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder that results from accelerating platelet clearance, destruction, and production. Front-line therapy for newly diagnosed ITP includes corticosteroids, intravenous immune globulin, or anti-D immunoglobulin. However, after these single-agent therapies, relapses will occur in half of patients. We previously reported the safety, feasibility, and efficacy of the combination of dexamethasone, low-dose rituximab, and the thrombopoietin receptor agonist (TPO-Ra) eltrombopag as front-line treatment in newly diagnosed ITP. Romiplostim, another TPO-Ra is approved by the FDA for patients with chronic ITP. However, the safety, tolerability, and efficacy of romiplostim combined with low-dose rituximab, and high-dose dexamethasone in newly diagnosed ITP remain unknown.

Background:Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease characterized by hemolysis and thrombosis. Many patients with PNH use C5-inhibitors (i.e., eculizumab/ravulizumab) to control their symptoms. Although C5-inhibition prevents intravascular hemolysis (IVH), it fails to prevent extravascular hemolysis (EVH). Because of persistent EVH, up to 72% of eculizumab-treated patients remain anemic, and up to 36% require at least one transfusion per year. Pegcetacoplan (PEG), a C3-inhibitor recently approved by the US FDA to treat adults with PNH, controls IVH and prevents EVH. Studies of PEG treatment in patients with PNH that remained anemic despite eculizumab treatment demonstrated that PEG was superior to eculizumab in achieving improvements in hemoglobin (Hb) levels (Hillmen P, et al., N Engl J Med, 2021 384 (11):1028-1037). Additionally, two early phase open-label trials demonstrated the efficacy of PEG in complement-inhibitor naïve patients with PNH (Wong RS, et al., Blood,2020 136 [Supplement 1]).