hydroxyurea

Ropeginterferon alfa-2b-njft (ropeg), a mono-PEGylated interferon-α, showed efficacy and safety in patients with hydroxyurea-intolerant/resistant essential thrombocythemia (ET) in the phase 3 SURPASS-ET largely conducted in Asia. Ropeg showed efficacy and substantial molecular responses with good overall tolerability across a broad ET population. PharmaEssentia.

P4, N=90, Completed, Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh | Recruiting --> Completed | Trial completion date: Dec 2024 --> Jan 2026 | Trial primary completion date: Jun 2024 --> Jan 2026

ORR was 57%, 20% and 25% in patients treated by intensive chemotherapy (IC), hypomethylating agents (HMA) and BSC (including low intensity treatments as hydroxyurea and low-dose cytarabine), respectively. We observed poor outcome using IC or HMA encouraging us to propose new clinical trials in this specific subgroup. Only ASCT was able to improve prognosis.

P2, N=50, Recruiting, Step Pharma, SAS | Phase classification: P1 --> P2 | N=20 --> 50 | Trial completion date: Dec 2027 --> Sep 2028 | Trial primary completion date: May 2026 --> Jun 2028

This study provides one of the first extensive characterizations of PV in southern Colombia, confirming internationally recognized clinical features, including advanced age at diagnosis, increased prevalence of cardiovascular comorbidities, and a predominance of high-risk classification. The low rate of finding JAK2 mutations suggests that molecular testing may not be as easy to get as it could be. Even if the treatment followed the guidelines, the risk of recurrence and thrombosis remained, showing that PV is a long-term and worsening condition. These findings highlight the urgent need to expand access to molecular diagnostics, develop tailored risk-adapted medicines, and initiate prospective multicenter studies in Latin America to optimize outcomes and quality of life in PV.

We describe a 71-year-old man with JAK2-positive polycythemia vera (PV) whose hematologic parameters remained suboptimally controlled under hydroxycarbamide treatment...This case demonstrates that additional mutated clones can significantly influence phenotype and disease evolution, highlighting the limitations of stepwise molecular testing. Comprehensive profiling of clonal architecture is essential to refine diagnosis and prognosis, in the NGS era.

Treatment strategies included supportive care, interferon, hydroxyurea, and ruxolitinib. PMF and SMF demonstrate distinct clinical phenotypes despite sharing bone marrow fibrosis as a common endpoint. The mutational landscape highlights the genetic heterogeneity of MF and underscores the importance of integrating clinical, pathological, and molecular information to improve disease characterization and guide individualized management.

P2/3, N=207, Active, not recruiting, Sobi Inc. | Recruiting --> Active, not recruiting

P2, N=107, Completed, Chia Tai Tianqing Pharmaceutical Group Co., Ltd. | Active, not recruiting --> Completed

Secondary prevention included dual antiplatelet therapy, therapeutic phlebotomy, and cytoreductive therapy with hydroxyurea...Long-term outcomes depend on integration of standard acute coronary syndrome therapy with disease-specific cytoreductive strategies to reduce recurrent thrombosis. This case reinforces that acute coronary syndromes should not be routinely attributed to atherosclerotic disease when clinical and angiographic features are disproportionate to traditional risk profiles.

P2, N=45, Not yet recruiting, St. Jude Children's Research Hospital

Modern treatment approaches, including therapeutic phlebotomy, low-dose aspirin, and cytoreductive therapies (hydroxyurea, interferon-α formulations, and ruxolitinib), have reduced blood cell counts and thrombotic risk, yet concerns about treatment-associated cardiovascular toxicity have emerged. Recent cardio-oncology guidelines emphasize structured monitoring for cancer therapy-related cardiovascular toxicity. This mini review summarizes the cardiovascular burden of PV, therapeutic strategies to mitigate risk, and emerging perspectives on cardiotoxicity-including a recently reported case of reversible left ventricular dysfunction associated with ropeginterferon α-2b.