ArticleLeukemia2015
Mutations and long-term outcome of 217 young patients with essential thrombocythemia or early primary myelofibrosis.
Article in Leukemia, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 2 of them syntheses that pooled it.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
29 citing papers in PubMed, 2 syntheses or guidelines pooled it, 70 citations in OpenAlex.
- Comparison of the effects between MPL and JAK2V617F on thrombosis and peripheral blood cell counts in patients with essential thrombocythemia: a meta-analysis.Annals of hematology · 2021Pooled it
- Characteristics and outcomes of patients with essential thrombocythemia or polycythemia vera diagnosed before 20 years of age: a systematic review.Haematologica · 2019Pooled it
- Cardiovascular Safety of Anagrelide Hydrochloride versus Hydroxyurea in Essential Thrombocythaemia.Cardiovascular toxicology · 2021Trial
- Mutant calreticulin enables potent and selective CAR-T cell therapy in preclinical models of myeloproliferative neoplasms.Journal for immunotherapy of cancer · 2026Article
- Clinical characteristics in adolescents and young adults with polycythemia vera and essential thrombocythemia in Japan.International journal of hematology · 2024Article
- FRACTION: protocol of a phase II study of Fedratinib and Nivolumab combination in patients with myelofibrosis and resistance or suboptimal response to JAK-inhibitor treatment of the German MPN study group (GSG-MPN).Annals of hematology · 2024Article
- Article
- Epidemiology and disease characteristics of myelofibrosis: a comparative analysis between Italy and global perspectives.Frontiers in oncology · 2024Review
- Young patients with myelofibrosis have distinct clinicomolecular features, favorable prognosis, and commonly exhibit inflammatory comorbidities.Annals of hematology · 2024Article
- Clinical features and next-generation sequencing landscape of essential thrombocythemia, prefibrotic primary myelofibrosis, and overt fibrotic primary myelofibrosis: a Chinese monocentric retrospective study.Journal of cancer research and clinical oncology · 2023Article
- Budd-Chiari syndrome in myeloproliferative neoplasms: A review of literature.World journal of clinical oncology · 2023Review
- [Clinical and genetic characteristics of young patients with myeloproliferative neoplasms].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Article
- Essential Thrombocythemia: One-Center Data in a Changing Disease.Medicina (Kaunas, Lithuania) · 2022Article
- Low-risk polycythemia vera and essential thrombocythemia: management considerations and future directions.Annals of hematology · 2022Review
- Essential Thrombocythemia in Children and Adolescents.Cancers · 2021Review
- Clinical Characteristics and Management of Cerebral Venous Sinus Thrombosis in Patients with Essential Thrombocythemia.Neuropsychiatric disease and treatment · 2021Article
- Primary myelofibrosis with concurrentWorld journal of clinical cases · 2020Article
- Myeloproliferative Neoplasms in Children, Adolescents, and Young Adults.Current hematologic malignancy reports · 2020Review
- Thrombophilic Risk of Factor V Leiden, Prothrombin G20210A, MTHFR, and Calreticulin Mutations in Essential Thrombocythemia Egyptian Patients.Advances in hematology · 2020Article
- Unfolding the Role of Calreticulin in Myeloproliferative Neoplasm Pathogenesis.Clinical cancer research : an official journal of the American Association for Cancer Research · 2019Review
Corrections and comments
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Authors and funding
18 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We investigated the influence of molecular status on disease characteristics and clinical outcome in young patients (⩽ 40 years) with World Health Organization (WHO)-defined essential thrombocythemia (ET) or early/prefibrotic primary myelofibrosis (early-PMF). Overall, 217 patients with ET (number 197) and early-PMF (number 20) were included in the analysis. Median follow-up time was 10.2 years. The cumulative incidence of thrombosis, hemorrhages and disease evolution into myelofibrosis/acute leukemia were 16.6%, 8.6% and 3% at 15 years, respectively. No differences were detectable between ET and early-PMF patients, although the latter cohort showed a trend for worse combined-event free survival (EFS). Mutation frequency were 61% for JAK2V617F, 25% for CALR and 1% for MPLW515K, and were comparable across WHO diagnosis; however, JAK2V617F allele burden was higher in the early-PMF group. Compared with JAK2V617F-positive patients, CALR-mutated patients displayed higher platelet count and lower hemoglobin level. CALR mutations significantly correlated with lower thrombotic risk (9.1% versus 21.7%, P = 0.04), longer survival (100% versus 96%, P = 0.05) and better combined-EFS (86% versus 71%, P = 0.02). However, non-type 1/type 2 CALR mutations ('minor' mutations) and abnormal karyotype were found to correlate with increased risk of disease evolution. At last contact, six patients had died; in five cases, the causes of death were related to the hematological disease and occurred at a median age of 64 years (range: 53-68 years). Twenty-eight patients (13%) were unmutated for JAK2, CALR and MPL: no event was registered in these 'triple-negative' patients.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.