hydroxyurea

P2, N=50, Not yet recruiting, PETHEMA Foundation

Treatment with hydroxycarbamide and ruxolitinib resulted in decreased platelet counts and improved vasculitis, with no subsequent recurrence of cardiovascular events. This rare case shows that ruxolitinib can be effective in treating vasculitis complications in patients with JAK2 mutation-positive ET.

The patient received cytoreductive therapy with hydroxycarbamide, combined with dual antiplatelet therapy...Recently, JAK2 V617F mutation in vascular endothelial cells of patients with MPN has been documented, suggesting a potential association between MPN and arterial dissection or dissecting aneurysm. Although rare, such vascular complications can be life-threatening and should be recognized as clinically significant.

The patient underwent therapeutic phlebotomy shortly after angioplasty and was subsequently started on hydroxyurea to maintain a hematocrit below 45%, together with dual antiplatelet therapy. This case highlights acute myocardial infarction as a rare initial presentation of polycythemia vera. It underscores the importance of considering polycythemia vera in patients presenting with acute coronary syndrome and unexplained erythrocytosis, while acknowledging that, in the absence of intracoronary imaging, a definitive causal link between PV and the coronary event cannot be established.

Cytoreduction with hydroxyurea is key to secondary prevention. Current ACS guidelines do not specifically address ET-associated ACS, underscoring the need for further studies in this high-risk population.

Adults with hydroxyurea-resistant/intolerant PV were randomized 1:1 to ruxolitinib or BAT; crossover to ruxolitinib was allowed after primary analysis. Patients receiving ruxolitinib experienced decreased JAK2V617F allele burden, durable hematocrit control, and better symptom improvements versus BAT, reinforcing ruxolitinib clinical benefit. RESPONSE: https://clinicaltrials.gov/study/NCT01243944; RESPONSE-2: https://clinicaltrials.gov/study/NCT02038036.

SHMT depletion in RasV12DlgRNAi cells causes DNA and chromosome damage and renders these cells sensitive to genotoxic stressors such as X-rays or hydroxyurea...Taken together, our data suggest that a diminished SHMT activity may drive the progression of RasV12DlgRNAi cancers through ROS-induced genome instability. Additionally, our study points to a novel gene-nutrient interaction, SHMT-PLP, that impacts cancer growth with potential therapeutic implications.

Ongoing treatment with hydroxyurea was substituted with ropeg (week 0: 250 mcg; week 2: 350 mcg; week 4 onwards: 500 mcg every 2 weeks). In conclusion, ropeg was safe and induced CHCR associated with significant molecular responses in patients with early MF. ClinicalTrials.gov Identifier: NCT04988815.

All TKIs are not recommended during the first trimester due to their risk of teratogenesis, but imatinib and nilotinib may be cautiously used from Weeks 16-18 onward. Non-TKI therapies, such as hydroxyurea and interferon-α, are considered safe throughout pregnancy. Data on ponatinib and asciminib remain insufficient to allow the safe use of these agents during pregnancy. Future research should aim to improve treatment-free remission rates through novel agents and combination strategies to allow a higher proportion of younger patients to discontinue therapy. Clinicians should always counsel women on pregnancy risks during therapy while reassuring male patients of TKI safety when fathering children.

These results reveal a relatively homogeneous epidemiological profile across the Latin American region and underscore the need for more multicenter studies to better characterize pH- MPNs in Ecuador and the region, to optimize diagnostic and treatment strategies.

P2, N=70, Not yet recruiting, Uma Borate | Initiation date: Jan 2026 --> Apr 2026

P4, N=76, Terminated, Novartis Pharmaceuticals | Trial completion date: Jan 2027 --> Feb 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jan 2027 --> Feb 2026; Sponsor Decision