^
Contact us  to learn more about
our Premium Content:  News alerts, weekly reports and conference planners
DRUG CLASS:

Ribonucleotide reductase inhibitor

1m
Extensive bilateral pulmonary embolism revealing iron deficiency-masked JAK2-positive polycythemia vera: A case report. (PubMed, Radiol Case Rep)
The patient received therapeutic anticoagulation, low-dose aspirin, and hydroxyurea. This case highlights the role of multimodal imaging in diagnosis and risk stratification, and the need to consider masked polycythemia vera in unprovoked pulmonary embolism with unexplained microcytosis and elevated red blood cell count.
Journal
|
ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • JAK2 (Janus kinase 2)
|
hydroxyurea • aspirin
1m
Treatment of myeloproliferative neoplasms: Exploring new horizons of who and when to cytoreduce in patients with polycythemia vera and essential thrombocytosis. (PubMed, Semin Hematol)
In this evidence-based review, we trace the evolution of risk stratification in PV and ET and examine the clinical evidence supporting current cytoreductive options, specifically hydroxyurea, interferons, ruxolitinib, and anagrelide. We argue for a paradigm shift away from binarily driven thrombosis-centric risk stratification toward a personalized, proactive approach that integrates molecular and inflammatory biomarkers, expands the population offered cytoreduction, and prioritizes disease-modifying therapies. Prospective studies are needed to validate the incorporation of these markers into risk-adapted algorithms and to define the long-term impact of early disease modification on transformation, survival, and quality of life.
Journal
|
JAK2 (Janus kinase 2)
|
Jakafi (ruxolitinib) • hydroxyurea
1m
Hidden in Plain Sight: Systemic Mastocytosis Manifesting as Isolated Hepatosplenomegaly in the Absence of Cutaneous and Classical Manifestations-A Case Report and Literature Review. (PubMed, Clin Case Rep)
The patient was treated with cladribine (40 mg over five days) followed by maintenance hydroxyurea (300 mg twice daily), with significant clinical improvement at eight-week follow-up. This case underscores that SM-AHN can present with isolated hepatosplenomegaly and profound leukocytosis without cutaneous signs, and highlights the critical role of integrated molecular profiling, including KIT mutation analysis, in the diagnostic workup of atypical hematologic presentations.
Journal
|
ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • KIT (KIT proto-oncogene, receptor tyrosine kinase)
|
cladribine • hydroxyurea
1m
Thrombocytapheresis as a Bridge Intervention in JAK2-Mutant Myeloproliferative Neoplasm Complicated by Acquired von Willebrand Disease: A Case Report. (PubMed, J Clin Apher)
We describe a 74-year-old woman with JAK2V617F-mutated myeloproliferative neoplasm and hydroxyurea intolerance who presented with active mucosal bleeding and a platelet count of 952 000/μL...Repeat testing at 24 h showed improvement in vWF:RCo to 0.48 IU/mL (ratio 0.68), which likely reflects restoration of functional high-molecular-weight multimers. In this single case, thrombocytapheresis provided rapid and effective platelet reduction for AvWD secondary to myeloproliferative neoplasms when pharmacological cytoreduction is inadequate.
Journal • JAK2V617F
|
JAK2 (Janus kinase 2)
|
hydroxyurea
1m
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia (clinicaltrials.gov)
P=N/A, N=240, Not yet recruiting, Riphah International University
New trial
|
HBB (Hemoglobin Subunit Beta)
|
thalidomide • hydroxyurea
1m
Isolated Extreme Thrombocytosis as the Presenting Feature of Chronic Myeloid Leukemia-An Unusual Phenotype Treated With Plateletpheresis. (PubMed, R I Med J (2013))
She was started on hydroxyurea, but her platelet count continued to rise, requiring hospitalization for close monitoring, where she was initiated on dasatinib, a tyrosine kinase inhibitor (TKI). As her platelets peaked above 4,000 × 109/L (Reference Range [RR]: 150-450 × 109/L), given the high risk of both thrombotic and bleeding complications, she underwent plateletpheresis, which effectively reduced counts before TKI therapy could achieve disease control. This case highlights the importance of considering the diagnosis of CML in patients with isolated thrombocytosis, particularly in women, and emphasizes the role of plateletpheresis as a potential preventive bridge until TKI therapy takes effect.
Journal
|
ABL1 (ABL proto-oncogene 1)
|
ABL1 fusion
|
dasatinib • hydroxyurea
2ms
Hydroxyurea-associated digital gangrene: a case report and narrative review of reported cases and emerging pathophysiology. (PubMed, Thromb J)
Our literature review identified three previously reported cases of HU-associated digital gangrene, though limited to the lower extremities - two in chronic myeloid leukemia (CML) and one in sickle cell disease (SCD). In each case, gangrene developed after prolonged HU exposure, alternative etiologies were not substantiated, and stabilization or clinical improvement followed HU withdrawal. The present case aligns with this pattern while extending the reported phenotype to well-controlled PV and upper-extremity digits. Given the small number of reported cases, the pathophysiology remains incompletely defined and is largely extrapolated from studies of more frequently described HU-associated ulceration, histopathologic reports of HU-related tissue injury, and in vitro studies of HU effects on endothelial and circulating cells. Plausible mechanisms include cumulative endothelial injury, localized thrombo-occlusive microvascular dysfunction, impaired vascular and cutaneous repair, and interaction with PV-related microvascular susceptibility. Clinicians should include HU-associated vasculopathy in the differential diagnosis of otherwise unexplained digital ischemia, as prompt drug cessation may limit progression and improve digit salvage.
Journal
|
JAK2 (Janus kinase 2)
|
hydroxyurea
2ms
HELPFUL: Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation (clinicaltrials.gov)
P=N/A, N=200, Recruiting, Children's Hospital Medical Center, Cincinnati | Trial completion date: Dec 2026 --> Dec 2030 | Trial primary completion date: Dec 2025 --> Dec 2028
Trial completion date • Trial primary completion date
|
hydroxyurea
2ms
Efficacy of Combination of Hdroxyurea and Thalidomide Over Hydroxyurea or Thalidomide in the Treatment of Transfusion Dependent Thalassemia in Children (clinicaltrials.gov)
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
Trial completion • Trial completion date • Trial primary completion date
|
thalidomide • hydroxyurea
2ms
Limited prognostic value of ELN classification and relevance of molecular ontogeny in acute myeloid leukemia post myeloproliferative neoplasms: a retrospective multicenter study. (PubMed, Acta Haematol)
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.
Clinical • Retrospective data • Journal
|
JAK2 (Janus kinase 2) • SRSF2 (Serine and arginine rich splicing factor 2)
|
SRSF2 mutation
|
cytarabine • hydroxyurea
2ms
A real-world analysis of polycythemia vera at two comprehensive cancer centers in Cali, Colombia. (PubMed, Blood Cells Mol Dis)
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.
Journal • Real-world evidence
|
JAK2 (Janus kinase 2)
|
Jakafi (ruxolitinib) • hydroxyurea • aspirin
2ms
Enrollment open
|
IDH wild-type
|
Triapine (3-AP)