A Randomized, Multicenter Trial of Shorter Durations of Hypomethylating Agents in Lowe r Risk Myelodysplastic Syndromes 

Presenter: Ian Bouligny

• This randomized, multi-center trial evaluated abbreviated hypomethylating agent regimens in lower-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia. 
• Among 247 patients (median age 70.8 years), overall response rates (ORR) in transfusion-dependent cohorts were 53% for 3-day decitabine, 53% for 3-day azacitidine, and 48% for 5-day azacitidine. 
• Transfusion independence was achieved in 44%, 42%, and 40%, respectively. In transfusion-independent patients, ORR was highest with 5-day azacitidine (70%) vs 3-day decitabine (50%) and 3-day azacitidine (54%). 
• Five-day azacitidine improved event-free survival (HR 0.25–0.41) and overall survival (HR 0.13–0.46) compared to shorter regimens. Grade ≥3 thrombocytopenia occurred more with decitabine (45%).
• Shorter regimens are safe and effective in MDS, but 5-day azacitidine offers superior survival and safety balance, producing the most consistent EFS and OS benefit.

Reference 

Bouligny I et al, A randomized, multicenter trial of shorter durations of hypomethylating agents in lowerrisk Myelodysplastic Syndromes. 67th Annual Meeting of American Society of Hematology, 2025, abs25-13849.

Rituximab and Epcoritamab as First-line Therapy for Patients with High-tumor Burden Follicular Lymphoma: Results of a Multicenter Phase II Trial

Presenter: Reid Merryman

• This phase 2 trial evaluated rituximab (R) plus epcoritamab (Epcor), a CD3×CD20 bispecific antibody (BsAb), as time-limited first-line therapy in previously untreated grade 1–3A follicular lymphoma (FL) requiring treatment (stage II–IV, ECOG PS 0–2). 
• Thirty-five patients (median age 58; 97% stage III/IV; 57% FLIPI score 3–5) received 4 weekly doses of R followed by step-up Epcor dosing. 
• Best overall response rate (ORR) was 97% and complete metabolic response (CMR) was 91%, with 34/35 patients responding after 2 cycles. 
• 9-month progression-free survival (PFS) was 97%. Cytokine release syndrome (CRS) occurred in 43% overall but was lower with 3 step-up doses (11% vs 54%; p=0.03).
• Grade ≥3 infections occurred in 9%, and neutropenia in 20%.
• R+Epcor achieved rapid, deep, and durable responses with manageable safety.
• Rituximab debulking reduced CRS risk, supporting further evaluation of this chemotherapy-free regimen.

Reference

Merryman R et al, Rituximab and Epcoritamab as First-line Therapy for Patients with High-tumor Burden Follicular Lymphoma: Results of a Multicenter Phase II Trial. 67th Annual Meeting of American Society of Hematology, 2025, abs25-13849. 

Hetrombopag for the Treatment of Lower-risk Myelodysplastic Syndromes with Thrombocytopenia: A Prospective, Single-arm, Multicenter Study 

Presenter: Chen Mei

• In this prospective, single-arm, multicenter study evaluating hetrombopag in lower-risk myelodysplastic syndromes (LR-MDS) patients with severe thrombocytopenia, 41.9% (18 of 43 patients) achieved platelet responses, including 7.0% complete responses, with a median time to response of 7.0 weeks. 27.9% (12 patients) achieved platelet counts ≥50×10⁹/L.
• The treatment showed an acceptable safety profile, with treatment-emergent adverse events occurring in 30.2% of patients with infection being the most common (11.6%). 
• Grade 3-4 infections occurred in 9.3% of patients. suggesting hetrombopag may be a promising new therapeutic option, although further studies are warranted.
• Hetrombopag effectively increased platelet counts with an acceptable safety profile, showing promise as a therapeutic option for LR-MDS patients with thrombocytopenia.

Reference 

Mei C et al, Hetrombopag for the Treatment of Lower-risk Myelodysplastic Syndromes with Thrombocytopenia: A Prospective, Single-arm, Multicenter Study .67th Annual Meeting of American Society of Hematology, 2025, abs25-4279.

Pirtobrutinib vs Ibrutinib in Treatment-Naïve and Relapsed/Refractory CLL/SLL: Results from the First Randomized Phase III Study Comparing a Non-Covalent and Covalent BTK Inhibitor

Presenter: Jennifer Woyach

• This randomized phase 3 trial compared pirtobrutinib (200 mg QD) with ibrutinib (420 mg QD) in N=662 cBTKi-naïve CLL/SLL patients, including treatment-naïve (TN, n=225) and relapsed/refractory (R/R, n=437) cohorts. 
• Median age was 67 years; del(17p) was present in ~15%. 
• The primary endpoint—noninferiority of overall response rate (ORR)—was met: ITT ORR was 87.0% for pirtobrutinib vs 78.6% for ibrutinib (ORR ratio 1.11; p<0.0001), and R/R ORR was 84.0% vs 74.8% (ORR ratio 1.12; p<0.0001). 
• TN patients achieved ORR of 92.9% vs 85.8%, respectively. ORR consistently favored pirtobrutinib across subgroups in both the ITT and R/R population, including del(17p). 
• Progression-free survival (PFS) also trended in favor of pirtobrutinib across ITT (HR 0.57), R/R (HR 0.73), and TN (HR 0.24) populations, with 18-month PFS rates of 86.9% vs 82.3% (ITT) and 95.3% vs 87.6% (TN). 
• Overall survival showed no detriment (HR 0.961). 
• Safety profiles were comparable, but atrial fibrillation/flutter (2.4% vs 13.5%) and hypertension (10.6% vs 15.1%) were lower with pirtobrutinib. 
• Dose reductions and discontinuations for progression were less frequent with pirtobrutinib. 
• Treatment remains ongoing in 81.3% of pirtobrutinib and 69.5% of ibrutinib patients. 
• Pirtobrutinib demonstrated non-inferiority of ORR vs ibrutinib in both ITT and R/R populations.
• PFS, while not yet mature, trended in favor of pirtobrutinib, with the most pronounced effect in the TN population, which had the longest follow-up at this data cut.

Reference 

Woyach J et al, Pirtobrutinib vs Ibrutinib in Treatment-Naïve and Relapsed/Refractory CLL/SLL: Results from the First Randomized Phase III Study Comparing a Non-Covalent and Covalent BTK Inhibitor. 67th Annual Meeting of American Society of Hematology, 2025, abs25-2587. 

Primary Analysis of the SMART STOP Trial: Lenalidomide, Tafasitamab, Rituximab, and Acalabrutinib Alone and with Combination Chemotherapy in Newly Diagnosed Diffuse Large B-Cell Lymphoma

Presenter: Jason Westin

• The SMART STOP trial evaluated chemotherapy reduction or elimination in newly diagnosed large B-cell lymphoma (LBCL) using targeted therapy. 
• Sixty-one patients (median age 62; 76% advanced stage; 56% poor-risk R-IPI) received 21 day (d) cycles with lenalidomide (L) 25mg on d1-10, tafasitamab (T) 12mg/kg IV on d1, 8, and 15, rituximab (R) 375 mg/m² IV on d1, and acalabrutinib (A) 100mg orally twice/d (LTRA) followed by PET/CT-guided adaptation. 
• Median follow up was 22 months for survival. 
• After four cycles, overall response rate (ORR) was 90%, and complete response (CR) was 57%. 
• At end of therapy, CR reached 96.7% overall, including 100% in patients with early CR. 
• Patients achieving early CR received reduced chemotherapy: 35 patients (57%) had ≤2 CHOP cycles or none. 
• In Cohort 1 (2 CHOP cycles), 30-month progression-free survival (PFS) was 100%; in Cohort 2 (no CHOP), 18-month PFS was 91.7%. 
• Overall cohort PFS and OS at 24 months were 85% and 98%, respectively.
• Toxicity was manageable; rash occurred in 41% (grade 3 in 7%), and lenalidomide dose reduction was required in 57%. 
• These results demonstrate that chemotherapy reduction or removal is feasible without compromising efficacy in newly diagnosed LBCL who achieve a complete response after targeted therapy, supporting LTRA as a highly effective initial regimen for LBCL. 
• The combination of lenalidomide, tafasitamab, rituximab, and acalabrutinib is highly effective as an initial chemotherapy-free combination in patients with newly diagnosed LBCL.
• Further investigation in multicenter randomized trials is warranted.

Reference

Westin J et al, Primary Analysis of the Smart Stop Trial: Lenalidomide, Tafasitamab, Rituximab, and Acalabrutinib Alone and with Combination Chemotherapy in Newly Diagnosed Diffuse Large B-Cell Lymphoma. 67th Annual Meeting of American Society of Hematology, 2025, abs25-10609.

Benefits of Hydroxyurea in Hemoglobin SC: Results of the Open-Label Phase of the Prospective Identification of Variables as Outcomes for Treatment (PIVOT) Trial

Presenter: Luke Smart

• PIVOT is a Phase 2, double-blind, placebo-controlled trial evaluating hydroxyurea in children and adults (ages 5–50) with hemoglobin SC (HbSC) disease in Ghana. 
• After 12 months of blinded treatment, participants entered a 12-month open-label hydroxyurea phase. 
• Of N=212 randomized patients, n=196 enrolled in the open-label phase (93 hydroxyurea; 92 placebo) and completed ~175 patient-years of treatment. 
• Hydroxyurea dosing averaged ~18–19 mg/kg/day. 
• Dose-limiting toxicities (DLTs), mainly mild neutropenia or thrombocytopenia, occurred in 23% during open-label treatment versus 33% in the original hydroxyurea arm and 26% in the original placebo arm. 
• Laboratory benefits—hemoglobin 11.2 g/dL, MCV 91 fL, HbF 10%—were sustained in the hydroxyurea arm and achieved by the placebo arm after crossover. 
• Clinical benefits included reduced vaso-occlusive pain (0.56 vs 1.51 → 0.55 events/patient-year; IRR 0.36, p<0.0001), malaria (0.20 vs 0.29 events/patient-year), and hospitalizations (0.11 vs 0.23 events/patient-year). 
• Transfusion rates remained low. By Month 24, both arms had equivalent hematologic and clinical improvements. 
• These open-label data confirm the blinded phase PIVOT data and document that hydroxyurea is associated with tolerable DLT and sustained disease-modifying clinical benefits for both children and adults with HbSC.
• Hydroxyurea was well tolerated; provided durable and sustained disease-modifying benefits, supporting its use in HbSC disease and informing a planned Phase 3 trial. 

Reference 

Luke Smart et al, Benefits of Hydroxyurea in Hemoglobin SC: Results of the Open-Label Phase of the Prospective Identification of Variables as Outcomes for Treatment (PIVOT) Trial. 67th Annual Meeting of American Society of Hematology, 2025, abs25-10580.

First Results of the Phase III GIMEMA ALL2820 Trial Comparing Ponatinib Plus Blinatumomab to Imatinib and Chemotherapy for Newly Diagnosed Adult PH+ Acute Lymphoblastic Leukemia Patients

Presenter: Sabina Chiaretti

• The GIMEMA ALL2820 phase III trial compared an experimental chemo-free regimen (ponatinib 45/30 mg, as per age plus at least 2 cycles of IV blinatumomab) versus TKI, imatinib 800/600 mg daily, as per age) plus chemotherapy (6/4 cycles, as per age) as the control arm in newly diagnosed adult Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL; n = 236)
• Complete hematologic remission was higher in the experimental arm vs. control arm (94.4% vs 79.4%, p=0.001). 
• MRD negativity at day +133 was significantly greater with the chemo-free approach (70.2% vs 52.7%, p=0.009), rising to 80.3% after additional blinatumomab cycles.
•  Event-free survival at 18 months favored the experimental arm (89.9% vs 76.8%, p=0.011).
• This head-to-head comparison trial showed for the first time a significant advantage of a chemo-free targeted/immunotherapeutic strategy over a tyrosine kinase inhibitor/chemotherapy approach, supporting a chemo-free approach as the new standard for adult Ph+ ALL.

Reference

Chiaretti S et al, First results of the Phase III GIMEMA ALL2820 trial comparing ponatinib plus blinatumomab to imatinib and chemotherapy for newly diagnosed adult ph+ acute lymphoblastic leukemia patients. 67th Annual Meeting of American Society of Hematology, 2025, abs25-10580.

Ropeginterferon Alfa-2b in Essential Thrombocythemia of All Risk Levels Ineligible for Standard Cytoreduction: 12-Month Primary Endpoint Analysis from the ROP-ET Phase 3 Study

Presenter: Jean-Jacques Kiladjian

• The ongoing phase 3 ROP-ET trial evaluated ropeginterferon alfa-2b, a next-generation mono-pegylated interferon, in essential thrombocythemia. 
• This 3-year, single-arm study enrolled 132 adults requiring cytoreduction but intolerant, resistant or ineligible for standard agents.
•  Ropeginterferon alfa-2b (125μg) was administered subcutaneously every two weeks, with dose escalation to 250μg and 500μg if needed to achieve hematologic response. 
• Most patients were previously treated with hydroxyurea or anagrelide. 
• The secondary endpoints were molecular response, quality of life, safety, and long-term outcomes.
• Baseline median platelet count was 579×10⁹/L; driver mutations included Janus kinase 2 (59.8%) and calreticulin (27.9%). 
• Preliminary data showed good tolerability with only 3% discontinuations due to treatment emergent adverse events. 
• The primary endpoint (durable hematologic and clinical response at 12 months) will be analyzed later. 
• In this last-line population, treatment with ropeginterferon alfa-2b was well tolerated, with few discontinuations due to adverse events.
• Interim findings highlight ropeginterferon’s potential as a disease-modifying option for ET population.

Reference

Kiladjian AJ et al, Ropeginterferon Alfa-2b in Essential Thrombocythemia of All Risk Levels Ineligible for Standard Cytoreduction: 12-Month Primary Endpoint Analysis from the ROP-ET Phase 3 Study. 67th Annual Meeting of American Society of Hematology, 2025, abs25-2199. 

A Phase 2 Study of Canakinumab in Patient with Myelofibrosis: Results from Part 1

Presenter: Andrew Kuykendall

• In a phase 2 study, canakinumab, an IL-1β-neutralizing monoclonal antibody, was evaluated in 13 patients with relapsed/refractory myelofibrosis previously treated with or ineligible for JAK inhibitors. Patients received canakinumab 200 mg subcutaneously on day 1 of 21-day cycles. The primary endpoint was overall response rate (ORR) including CR, PR, or CI after 8 cycles (24 weeks). 
• At week 24, 23% patients achieved consistent clinical improvement in symptoms with canakinumab, while 46% progressed due to spleen volume increase.
•  Median hemoglobin improved from 8.2 g/dL to 9.0 g/dL, and platelet counts rose from 114×10⁹/L to 183×10⁹/L. 
• Symptom improvement (Myelofibrosis Symptom Assessment Form Total Symptom Score percent change from baseline range -20% to -85%) was reported in 85% patients. 
• Canakinumab lowered CRP by 37% in 62.5% of patients with baseline hsCRP ≥1.0.
• Adverse events were mainly disease-related cytopenias (anemia and thrombocytopenia); non-hematologic grade ≥3 events were rare and not related to canakinumab (except for one: an increase in bilirubin). 
• The IL-1β inhibitor, canakinumab, resulted in consistent symptom improvement and exhibited beneficial impact on cytopenias in pts with R/R MF while demonstrating a manageable toxicity profile. 

Reference

Kuykendall A et al, A Phase 2 Study of Canakinumab in Patient with Myelofibrosis: Results from Part 1. 67th Annual Meeting of American Society of Hematology, 2025, abs25-14615.

Tucidinostat plus R-CHOP in Untreated MYC/BCL2 Double-Expressor Diffuse Large B-Cell Lymphoma: Topline Efficacy and Safety Results from The Randomized Phase 3 DEB Study

Presenter: Wei Li Zhao

• Epigenetic dysregulation drives diffuse large B-cell lymphoma (DLBCL). Tucidinostat, a selective HDAC inhibitor, was tested in the Phase 3 DEB trial in previously untreated double-expressor lymphoma (DEL). 
• A total of 423 patients were randomized (211 tucidinostat + R-CHOP; 212 placebo + R-CHOP). 
• At 44.9-month median follow-up, tucidinostat reduced risk of progression/death by 28% (HR 0.72; 95% CI 0.54–0.96; P=0.02). 
• Event-free survival rates: 2-year 60.3% vs 50.5%; 3-year 56.8% vs 47.7%. Complete response rate: 73.0% vs 61.8% (difference 11.1%; P=0.01). 
• Safety was manageable; grade ≥3 hematologic AEs were higher with tucidinostat but no unexpected toxicities. 
• In previously untreated patients with DEL, Tucidinostat + R-CHOP significantly improves EFS and CR rates.

Reference

Zhao WL et al, Tucidinostat plus R-CHOP in Untreated MYC/BCL2 Double-Expressor Diffuse Large B-Cell Lymphoma: Topline Efficacy and Safety Results from The Randomized Phase 3 DEB Study. 67th Annual Meeting of American Society of Hematology, 2025, abs25-10603.

Primary Phase 3 Results from The Epcore FL-1 Trial of Epcoritamab with Rituximab and Lenalidomide (R2) versus R2 for Relapsed or Refractory Follicular Lymphoma

Presenter: Lorenzo Falchi 

• Relapsed/refractory follicular lymphoma (FL) has limited second line options; EPCORE FL-1 compared Epcoritamab + Rituximab + Lenalidomide (E+R²) vs R² alone in 488 patients (243 vs 245). Median age: 60 vs 63; follow-up: 10.4 months. 
• ORR was 95.7% (95% CI: 90.2–98.6) with E+R² vs 81.0% (95% CI: 72.7–87.7; P<.0001). CR rate: 74.5% vs 43.3% (P<.0001). 
• 12-month DOR: 91.4% vs 57.0%. PFS improved significantly (HR 0.21; 95% CI: 0.13–0.33; P<.0001), reducing progression/death risk by 79%. 
• CRS was low grade (24.4%; mostly G1/G2) and resolved; ICANS rare (1 case).
• G3/4 TEAEs: 88.1% vs 62.2%, mainly neutropenia (66.3% vs 37.8%) and infections (29.2% vs 13.4%). 
• Fatal TEAEs: 0.8% vs 2.9%.
• E+ R² improves ORR, CR, and PFS significantly in FL; sets a new benchmark as a readily available treatment, is suitable for outpatient administration, and has the potential to become a new standard of care in 2L+ FL.

Reference

Falchi L et al, Primary Phase 3 Results from The Epcore FL-1 Trial of Epcoritamab with Rituximab and Lenalidomide (R2) versus R2 for Relapsed or Refractory Follicular Lymphoma. 67th Annual Meeting of American Society of Hematology, 2025, abs25-244.

Golcadomide (GOLCA), A Potential, First-In-Class, Oral CELMoD™ Agent, plus R-CHOP in Patients (Pts) with Previously Untreated Aggressive B-Cell Lymphoma (a-BCL): 24-Month Efficacy Results

Presenter: Grzegorz Nowakowski 

• Approximately 40% of DLBCL patients relapse after first-line therapy, especially those with high-risk features. GOLCA, an oral CELMoD, was evaluated with R-CHOP in 78 untreated aggressive B-cell lymphoma patients (recommended dose: 0.4 mg D1–7). 
• At median 24-month follow-up, end-of-treatment complete metabolic response (CMR) was 88% (29/33) at 0.4 mg; high-risk patients had CMR 89%. MRD negativity reached 90%. 
• The 24-month PFS was 79% overall and in high-risk patients; OS was 85.3%.
• Responses were consistent across GCB (88%) and non-GCB (73%) phenotypes.
• Grade 3/4 hematologic AEs occurred in 91% (neutropenia 87%, thrombocytopenia 42%, anemia 36%), but were predictable and manageable.
• GOLCA + R-CHOP delivers durable CMRs and strong 24-month PFS. This data continues to show that GOLCA 0.4mg, when added to SOC Tx, has a potential to cure more previously untreated pts with HR LBCL and support the ongoing Phase 3 trial, GOLSEEK-1, in this population

Reference

Nowakowski G et al, Golcadomide (GOLCA), A Potential, First-In-Class, Oral CELMoD™ Agent, plus R-CHOP in Patients (Pts) with Previously Untreated Aggressive B-Cell Lymphoma (a-BCL): 24-Month Efficacy Results. 67th Annual Meeting of American Society of Hematology, abs25-8393.

Real-World Effectiveness and Safety of Hydroxyurea in Adults with Hemoglobin SC Disease: A Retrospective Cohort Study in Quebec, Canada

Presenter: Alice Girard

• This retrospective cohort study evaluated the treatment with hydroxyurea (HU) in 263 adults with hemoglobin SC disease (HbSC). 
• Of these, 110 were included in the security analysis and 75 in the efficacy analysis (women; 51.8%, mean age of 33 years, baseline Hb; 118 g/L, alpha thalassemia present in 37.3%, G6PD deficiency seen in 7.3%). 
• The median HU dose administered during the study was 17 mg/kg.
• HU led to a 56% reduction in annual vaso-occlusive composite crises (VOCC) from 0.57 to 0.25 (Δ = -0.32; 95% CI: -0.51 to -0.13; p = 0.0015). 
• Greater benefit was seen in patients aged 18–30 (-0.51), in those without G6PD deficiency (-0.31), and in those with alpha-thalassemia (-0.41). 
• The most common hematological AE was thrombocytopenia (32.7%), followed by neutropenia (23.6%), and leukopenia (20.9%), mostly grade 1.
• Liver enzyme elevations were the most frequent non-hematologic AE. 
• No hyperviscosity-related complications were confirmed. 
• Treatment discontinuation occurred in 27.3%, often due to lack of prescription renewal awareness or mild AEs. 
• HU showed meaningful efficacy - reduction in VOCC and tolerability, supporting its use in HbSC and highlighting the need for improved adherence strategies.

Reference

Girard A et al, Real-World Effectiveness and Safety of Hydroxyurea in Adults with Hemoglobin SC Disease: A Retrospective Cohort Study in Quebec, Canada 67th Annual Meeting of American Society of Hematology, abs25-11660.

Initial Results of the First-in-Human Phase 1 Study of IBI3003, a Novel Trispecific Antibody Targeting GPRC5D, BCMA and CD3, in Patients with Relapsed or Refractory Multiple Myeloma

Presenter: Jing Li

• This ongoing first-in-human phase 1 study evaluates the safety and efficacy of IBI3003, a trispecific antibody targeting GPRC5D, BCMA, and CD3 in patients with relapsed or refractory multiple myeloma (R/R MM, n=28). 
• Median age of the study population was 61.5 years (women; 53.6%, ECOG PS 1: 85.7%; R-ISS stage III: 32.1%; mSMART high-risk: 75.0%; median lines of prior treatments: 5; EMD: 50.0%; ≥60% plasma cells in bone marrow: 17.9%; triple-class exposed;100%, penta-class exposed; 53.6%). 
• IBI3003 was administered weekly at doses from 0.1 to 540 μg/kg. Overall response rates (ORR) were 84.6% at ≥120 μg/kg and 100% at ≥360 μg/kg. 
• sBCMA levels dropped by 72.15% after Cycle 1 at 120 μg/kg and 95.12% after Cycle 2 at 360 μg/kg. 
• Grade ≥3 adverse events occurred in 78.6%, mainly cytopenias and Grade 1–2 cytokine release syndrome (71.4%). 
• IBI3003 showed promising efficacy (across 40-540 μg/kg, including 100% ORR in patients receiving ≥ 360 μg/kg) and manageable safety in high-risk R/R MM, including those with prior anti-BCMA/GPRC5D therapy. 

Reference

Li J et al, Initial Results of the first-in-human Phase 1 study of IBI3003, a novel trispecific antibody targeting GPRC5D, BCMA and CD3, in patients with relapsed or refractory multiple myeloma. 67th Annual Meeting of American Society of Hematology, abs25-10881.

Safety and Efficacy of Ten Years of Hydroxyurea Treatment for Young Children with Sickle Cell Anemia in Uganda

Presenter: Russell Ware

• The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) trial enrolled 207 children from Uganda with sickle cell anemia (SCA; mean age 2.3 years) to assess the safety and efficacy of hydroxyurea (HU). 
• Initial fixed-dose HU (20 mg/kg/day) showed no increased malaria risk and yielded clinical benefits. 
• In the follow-on NOHARM-MTD trial dose escalation to ~30 mg/kg/day significantly improved outcomes. 
• The hemoglobin (Hb) levels improved to 8.6 g/dL, fetal Hb (HbF) improved to 26.3%, MCV was 99 fL, ANC was 3.3 x 10⁹/L, and platelets increased to 439 x 10⁹/L.
• Dose-limiting toxicities (DLT) were infrequent and typically occurred during a febrile illness. 
• Over 1808 patient-years, maximum tolerated dose (MTD) reduced serious adverse events (IRR: 0.37, p = 0.011), hospitalizations (IRR: 0.19, p < 0.0001), and mortality (IRR: 0.27, p = 0.018). 
• Stroke incidence was low (0.11/100 patient-years). Serial transcranial Doppler (TCD) screening revealed normal velocities in 93.3% of 1426 completed scans, conditional velocities in 6.3%, and abnormal velocities in 0.5% of scans. 
• Current cohort (n = 175) averages 12.6 years old and demonstrates sustained safety and efficacy of the medication. 
• These findings support early, optimized HU use to improve long-term outcomes in this patient population in low-resource settings.

Reference 

Ware R et al, Safety and Efficacy of Ten Years of Hydroxyurea Treatment for Young Children with Sickle Cell Anemia in Uganda.   67th Annual Meeting of American Society of Hematology, abs25-14786.