Blood Advances

Single-agent rituximab can be a suitable treatment in hairy cell leukemia (HCL) patients relapsing after repeated courses of purine analogs, if purine analogs are contraindicated (e.g. in case of poor marrow cellularity, high disease infiltration predicting long-lasting aplasia) and if newer agents (moxetumomab or vemurafenib) are not easily available. Our institutional series of patients receiving single-agent rituximab as salvage therapy was reviewed. Patients received rituximab at the standard dose of 375 mg/m2 weekly for 4 weeks...

Hemophagocytic Lymphohistiocytosis (HLH) and Macrophage Activation Syndrome (MAS) are life-threatening hyperinflammatory syndromes. Familial HLH is caused by genetic impairment of granule-mediated cytotoxicity (e.g., perforin-deficiency). MAS is linked to excess activity of the inflammasome-activated cytokine IL-18. Though individually tolerated, mice with dual susceptibility (Prf1-/-Il18tg; DS) succumb to spontaneous, lethal hyperinflammation. We hypothesized that understanding how these susceptibility factors synergize would uncover key pathomechanisms in the activation, function, and persistence of hyperactivated CD8 T-cells...

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Hydroxyurea reduces sickle cell disease (SCD) complications, but medication adherence is low. We tested two mobile health (mHealth) interventions targeting determinants of low adherence among patients (InCharge Health) and low prescribing among providers (HU Toolbox) in a multi-center non-randomized trial of individuals with SCD ages 15-45. We compared the percentage of days covered (PDC), labs, healthcare utilization, and self-reported pain over 24 weeks of intervention and 12 weeks post-study with a 24-week pre-intervention interval...

The efficacy and safety of nilotinib in pediatric patients with imatinib/dasatinib resistant/intolerant (R/I) or newly diagnosed (ND) Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia in chronic phase (CML-CP) was demonstrated in the phase II, open-label DIALOG study. In this final analysis, long-term efficacy and safety are presented for patients who completed 66 cycles (of 28 days) of treatment with nilotinib (230 mg/m2 twice daily) or discontinued early. Fifty-nine patients were enrolled and 58 were treated (R/I, n=33; ND n=25; median time on treatment: 60...

Hodgkin lymphoma (HL) has become one of the most curable cancers. Therefore, rigorous assessment of health-related quality of life (HRQoL) and symptom burden of these patients is essential to support informed clinical decisions. This paper reports the final results of an international study by the European Organisation for Research and Treatment of Cancer (EORTC) to develop a HRQoL disease-specific measure for these patients: the EORTC QLQ-HL27. Patients with a confirmed diagnosis of HL (N=381) were enrolled from 12 countries and completed the EORTC QLQ-C30, QLQ-HL27 and a debriefing questionnaire at baseline (any time after diagnosis)...

Allogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). In young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of two TBI-free conditioning regimens in children with ALL <4 years old. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT...

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In pre-novel agent era, median post progression overall survival (PPS) of classic Hodgkin lymphoma (cHL) patients (pts) who progress after ASCT have been 2-3 years. Recently, Checkpoint inhibitors (CPI) and brentuximab vedotin (BV) have improved depth and durability of response in this population. Here we report estimate of PPS in pts with relapsed cHL after ASCT in the era of CPI and BV. In this multicenter retrospective study of 15 participating institutions, adult pts with relapsed cHL after ASCT were included...

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Thoracic radiation is associated with significant cardiopulmonary morbidities in long-term Hodgkin lymphoma (HL) survivors and may impact neurocognitive outcomes. Survivors (N=204, 52.5% female, mean[SD] age 36.6[8.01] years), treated with thoracic radiation, and age-, sex- and race/ethnicity-matched community controls (N=205, 51.7% female, age 36.7[9.17] years) completed standardized neurocognitive testing, echocardiography, pulmonary function tests, and vascular studies during the same visit. Treatments were abstracted from medical records...

Preexisting autoimmune disease affects 10-30% of patients with myelodysplastic syndromes (MDS). Studies comparing outcomes in MDS patients with and without autoimmune disease show discordant results. Using Surveillance, Epidemiology, and End Results (SEER)-Medicare, we conducted a population analysis to define the impact of autoimmunity on MDS outcomes. Cases were ascertained between 2007 - 2017 and claim algorithms employed to identify autoimmune disease, demographic characteristics, comorbidity scores, MDS histology, transfusion burden, treatment with hypomethylating agents and hematopoietic stem cell transplant...

Acute megakaryoblastic leukemia (AMKL) is a rare, developmentally restricted and highly lethal cancer of early childhood. The paucity and hypocellularity (due to myelofibrosis) of primary patient samples hamper the discovery of cell- and genotype-specific treatments. AMKL is driven by mutually exclusive chimeric fusion oncogenes in two thirds of cases, with CBFA2T3::GLIS2 (CG2) and NUP98 fusions (NUP98r) representing the highest fatality subgroups. We established CD34+ cord blood-derived CG2 models (n=6) that sustain serial transplantation and recapitulate human leukemia regarding immunophenotype, leukemia initiating cell frequencies, co-mutational landscape and gene expression signature with distinct upregulation of the pro-survival factor BCL2...

Hairy cell leukemia (HCL), like its variant HCLv, is a B-cell malignancy associated with decreased humoral immunity. We prospectively monitored the largest cohort of HCL/HCLv patients to date (n=503) for COVID-19 by symptoms, antibody, PCR and/or antigen-positivity. Fifty percent (253 of 503) of the HCL/HCLv patients (238 HCL, 15 HCLv) had evidence of COVID-19, with 210 (83%) of 253 testing positive by PCR or rapid-antigen. Of 43 without positive tests, all had nucleocapsid antibodies indicating COVID-19 exposure, 7 recalled no symptoms, and 36 had mild symptoms but without a positive test...

Hemogenic endothelial cells (HECs) are specialized cells that undergo endothelial to hematopoietic transition (EHT) to give rise to the earliest precursors of hematopoietic progenitors that will eventually sustain hematopoiesis throughout the lifetime of an organism. Although HECs are thought to be primarily limited to the aorta gonad mesonephros (AGM) during early development, EHT has been described in various other hematopoietic organs and embryonic vessels. Though not defined as a hematopoietic organ, the lung houses many resident hematopoietic cells, aids in platelet biogenesis, and is a reservoir for hematopoietic stem and progenitor cells (HSPCs)...

Investigating prognostic factors in relapsed or primary refractory classical Hodgkin lymphoma (R/R cHL) patients is essential to optimize risk-adapted treatment strategies. We built a prognostic model using baseline quantitative 18F-FDG PET radiomics features and clinical characteristics to predict progression free survival (PFS) in R/R cHL patients treated with salvage chemotherapy followed by autologous stem-cell transplant (ASCT). Metabolic tumor volume (MTV) and several novel radiomics dissemination features representing inter-lesional differences in distance, volume and standard uptake value (SUV) were extracted from the baseline PET...

The Virtual Opinions poll Independent Centered on CLL patients' Experience (VOICE) evaluated patients' knowledge about chronic lymphocytic leukemia (CLL), their perspectives on diagnosis and treatment, and their unmet needs. Clinicians and patient advocacy group representatives developed and distributed the survey from March through December 2022 in 12 countries, and 377 patients with ≥1 line of previous CLL treatment responded from Europe, Latin America, USA, Australia, Egypt, and Turkey. A large majority (90%; 336/374) relied on their physicians for information about CLL and treatment...

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