Contributions
Abstract: PB2287
Type: Publication Only
Background
Primary myelofibrosis (PMF) is characterized by the dysregulated proliferation of myeloid cells including megakaryocytes and myeloid and erythroid progenitors in the bone marrow (BM), resulting in ineffective erythropoiesis and the reactive deposition of fibrous connective tissue (reticulin/collagen) often with osteosclerosis. The related myeloproliferative neoplasms essential thrombocythemia (ET) and polycythemia vera (PV) can both undergo delayed disease transformation into a fibrotic state: post-ET myelofibrosis (post-ET MF) or post- PV MF, respectively. Individuals affected by MF face unique challenges, even compared to other MPN patients.
Aims
The main aim of this study is to evaluate clinical features and hematological parameters, of patients with PMF on ESAs. The secondary aim is to assess response rates and duration of response in terms of IWG-MRT.
Methods
This is a retrospective analysis of clinical and laboratory features, ESAs treatment modalities, and outcomes of PMF patients evaluated between July 2015 until December 2017. Continuous and categorical variables were tested as SPSS Statistics analysis: We conducted a review of the medical data of the patient on ESAs Respondents were recruited via pharmacy software data. In total, 11 patients with primary MF or MF post-thrombocythemia / polycythemia vera (post-ET / PV: n = 6) received ESAs in monotherapy for anemia (Hb <10g / dL). The concomitant use of cytoreduces treatment was allowed. According to criteria of the IWG-MRT, a favorable response is considered the cessation of the transfusion requirements in patients with transfusional dependence or the increase in Hb> 2g / dL in those who were not transfused, maintained for at least 12 weeks.
Results
Patients had a diagnosed of PMF approximately 2.5 years ago. Most (59.1%) were male and predominant Caucasian population (97.6%). A total of 11 patients (64.7%) obtained clinical improvement of the anemia, after a median of 3.8 months of treatment. In the univariate analysis, the baseline factors associated with a higher probability of response were: female gender, MF post-ET / PV, leukocytes > 10 x109/L, EPO levels <125 U / L, serum ferritin levels < 200 ng / mL, grade 1-2 marrow fibrosis and absence of cytogenetic alterations or previous transfusions. Based on the binary logistic regression analysis, the clinical factors with independent prognostic significance associated with the response were the patient's gender, the number of leukocytes and the ferritin levels. The median duration of the response was 18.2 months (IQR]: 9.5-21.4). After a median follow-up from the start of the 24-month treatment, 5 patients (29.4%) had discontinued ESAs.
Conclusion
The results of the study demonstrated that ESAs significantly improved the anaemia in about half of patients with MF and induced a durable response rates [risk ratio (RR): 2.77, 95% confidence interval (CI): 2.01–3.86, P = 0.03; RR: 7.52, 95% CI: 3.81–6.2, P = 0.05; respectively]. Some clinical factors can help predict the likelihood of response to treatment. An early initiation of ESAs could offer better results. The potential benefit is more significant in transfusion independent patient with baseline Haemoglobin levels of 90 g/L. A specific educational program needs to develop specifically for this population. The results of the research promote a development of comprehensive local guidelines.
Session topic: 16. Myeloproliferative neoplasms - Clinical
Keyword(s): Erythropoietin, Myelofibrosis, Myeloproliferative disorder
Abstract: PB2287
Type: Publication Only
Background
Primary myelofibrosis (PMF) is characterized by the dysregulated proliferation of myeloid cells including megakaryocytes and myeloid and erythroid progenitors in the bone marrow (BM), resulting in ineffective erythropoiesis and the reactive deposition of fibrous connective tissue (reticulin/collagen) often with osteosclerosis. The related myeloproliferative neoplasms essential thrombocythemia (ET) and polycythemia vera (PV) can both undergo delayed disease transformation into a fibrotic state: post-ET myelofibrosis (post-ET MF) or post- PV MF, respectively. Individuals affected by MF face unique challenges, even compared to other MPN patients.
Aims
The main aim of this study is to evaluate clinical features and hematological parameters, of patients with PMF on ESAs. The secondary aim is to assess response rates and duration of response in terms of IWG-MRT.
Methods
This is a retrospective analysis of clinical and laboratory features, ESAs treatment modalities, and outcomes of PMF patients evaluated between July 2015 until December 2017. Continuous and categorical variables were tested as SPSS Statistics analysis: We conducted a review of the medical data of the patient on ESAs Respondents were recruited via pharmacy software data. In total, 11 patients with primary MF or MF post-thrombocythemia / polycythemia vera (post-ET / PV: n = 6) received ESAs in monotherapy for anemia (Hb <10g / dL). The concomitant use of cytoreduces treatment was allowed. According to criteria of the IWG-MRT, a favorable response is considered the cessation of the transfusion requirements in patients with transfusional dependence or the increase in Hb> 2g / dL in those who were not transfused, maintained for at least 12 weeks.
Results
Patients had a diagnosed of PMF approximately 2.5 years ago. Most (59.1%) were male and predominant Caucasian population (97.6%). A total of 11 patients (64.7%) obtained clinical improvement of the anemia, after a median of 3.8 months of treatment. In the univariate analysis, the baseline factors associated with a higher probability of response were: female gender, MF post-ET / PV, leukocytes > 10 x109/L, EPO levels <125 U / L, serum ferritin levels < 200 ng / mL, grade 1-2 marrow fibrosis and absence of cytogenetic alterations or previous transfusions. Based on the binary logistic regression analysis, the clinical factors with independent prognostic significance associated with the response were the patient's gender, the number of leukocytes and the ferritin levels. The median duration of the response was 18.2 months (IQR]: 9.5-21.4). After a median follow-up from the start of the 24-month treatment, 5 patients (29.4%) had discontinued ESAs.
Conclusion
The results of the study demonstrated that ESAs significantly improved the anaemia in about half of patients with MF and induced a durable response rates [risk ratio (RR): 2.77, 95% confidence interval (CI): 2.01–3.86, P = 0.03; RR: 7.52, 95% CI: 3.81–6.2, P = 0.05; respectively]. Some clinical factors can help predict the likelihood of response to treatment. An early initiation of ESAs could offer better results. The potential benefit is more significant in transfusion independent patient with baseline Haemoglobin levels of 90 g/L. A specific educational program needs to develop specifically for this population. The results of the research promote a development of comprehensive local guidelines.
Session topic: 16. Myeloproliferative neoplasms - Clinical
Keyword(s): Erythropoietin, Myelofibrosis, Myeloproliferative disorder