An observational study in Myelofibrosis; Anemia, sponsored by Centre Hospitalier Annecy Genevois. Completed. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-07-20.
Sponsored by Centre Hospitalier Annecy Genevois · Observational
In patients with myelofibrosis, ruxolitinib is a current therapeutic option, which has demonstrated rapid and durable reduction in splenomegaly and improved disease-related symptoms. Anemia is another frequent issue in myelofibrosis. Consistent with its JAK2 signalling inhibition, ruxolitinib therapy has been shown to be detrimental on the hemoglobin level, increasing the depth of anemia or transfusion need.
Despite potential antagonistic mechanisms of action on Janus Kinase 2, some responses on anemia have been reported with the addition of Erythropoiesis-stimulating Agent to ruxolitinib in a small subset of patients in the COMFORT II study.
Ruxo-EPO is an observational study aimed to better assess the combination of Erythropoiesis-stimulating Agent and a Janus Kinase 2 inhibitor for therapeutic efficacy on anemia, Myelofibrosis evolution and for tolerance, in a larger cohort of patients treated for myelofibrosis in general practice.
Myeloproliferative Neoplasms are chronic diseases; they encompass Polycythemia Vera, Essential Thrombocythemia, and Primary Myelofibrosis with shared mutations that constitutively activate the physiologic signal-transduction pathways responsible for hematopoiesis. Myelofibroses may arise either as primary or antecedent to Polycythemia Vera and Essential Thrombocythemia. Myelofibrosis is the least common and the most aggressive of these diseases leading to death in 5 to 7 years due to bone marrow failure, massive splenomegaly related to extra medullary hematopoiesis, constitutional symptoms and cachexia.
Until recently, treatment of Myelofibrosis consisted of supportive care only, especially transfusions.
Since 2005 driver mutations have been identified in more than 90% of patients with myeloproliferative neoplasms, providing substantial insight into their pathogenesis and leading to the development of JAK inhibitor drugs. Ruxolitinib, the first one available, has shown activity whatever the presence of Janus Kinase 2 mutation, in reducing splenomegaly, improving cytopenias, alleviating constitutional symptoms and finally improving survival (COMFORT I and II studies). In France, access to ruxolitinib has been possible since April 2011, before wider availability in November 2012.
Anemia is a frequent issue in Myelofibrosis, which may be managed by the use of Erythropoiesis-stimulating Agent, leading to a 40-50% response rate in small studies.
Consistent with its Janus Kinase 2 signalling inhibition, ruxolitinib therapy has been shown to be detrimental on the hemoglobin level, increasing the depth of anemia or transfusion need, especially during the first 12-24 weeks of treatment in the COMFORT studies.
Despite potential antagonistic mechanisms of action on Janus Kinase 2, some responses on anemia have been reported with the addition of Erythropoiesis-stimulating Agent to ruxolitinib in a small subset of patients in the COMFORT II study.
Ruxo-EPO is an observational study aimed to better assess the combination of Erythropoiesis-stimulating Agent and a Janus Kinase 2 inhibitor for therapeutic efficacy on anemia, Myelofibrosis evolution and for tolerance, in a larger cohort of patients treated for myelofibrosis in general practice.
431 studies on the registry are indexed under Primary Myelofibrosis; 118 are open to participants now.
This study's enrollment of 65 is below the median of 134 across 56 observational studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →Centre Hospitalier Annecy Genevois is the lead sponsor of 32 studies on the registry; 8 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients with myelofibroses treated with a combination of Janus Kinase 2 Inhibitor and Erythropoiesis-stimulating Agent in one of the France Intergroup Myeloproliferative neoplasms centers
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Exclusion Criteria:
- patients who denied participating to this observational study
Effect on anemia of the combination Janus Kinase 2 Inhibitor and Erythropoiesis-stimulating Agent
Response rate assessed with 2006 International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European Leukemia Network (ELN) criteria
Time frame: 6 months
Patterns of the combination Janus Kinase 2 Inhibitor and Erythropoiesis-stimulating Agent
Modalities of the combination (drugs of interest begun at the same or at different dates, order of the drug combination if started at different dates, duration of combination, doses of each drug)
Time frame: 6 months
Patients and Myelofibrosis characteristics
Demographic characteristics of included patients, Characteristics of diseases (date of diagnosis, primary or secondary myelofibrosis, prognostic scores, level of hemoglobin, transfusion dependency, splenomegaly, mutations, delay between myelofibrosis diagnosis and Jak2 inhibitor treatment)
Time frame: at inclusion
Response rate of the combination treatment on constitutional symptoms of myelofibrosis
Time frame: 3 years
Response rate of the combination treatment on splenomegaly
Splenomegaly size assessment
Time frame: 3 years
Efficacy of the combination treatment on bone marrow fibrosis
Assessment of bone marrow fibrosis change if bone marrow biopsy has been done
Time frame: 3 years
Efficacy of the combination treatment on Janus Kinase 2 allelic burden change
Assessment of Janus Kinase 2 allelic burden change if molecular evaluation has been done in blood
Time frame: 3 years
Adverse Events on combination treatment
Time frame: 3 years
Overall survival on combination treatment
Time frame: 3 years
No study locations are listed for this record.
Plan to share: No
Ran from May 2016 to primary completion in Dec 2016, 7 months.
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This study is completed, as verified in Jul 2022. You cannot join it, but the record below documents what was studied.
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Centre Hospitalier Annecy Genevois