Improved survival of patients with myelofibrosis in the last decade: Single‐center experience

Masárová, Lucia; Bose, Prithviraj; Pemmaraju, Naveen; Daver, Naval; Sasaki, Koji; Chifotides, Helen T.; Zhou, Lingsha; Kantarjian, Hagop M.; Estrov, Zeev; Verstovšek, Srđan

doi:10.1002/cncr.34103

Cited by 23 publications

(11 citation statements)

References 35 publications

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“…Similar observations are emerging after long-term follow-up of other JAKI agents, such as the JAK1/ JAK2 and ACVR1 inhibitor momelotinib, suggesting a possible survival advantage in patients with newly diagnosed MF as well as RUXexposed patients. 15 Although the improvement in survival of patients with MF over the last decade 11 is likely multifactorial and attributable in part to advances in supportive care, taken together, our data, the pooled analyses of the COMFORT trials, 12 and the various registry experiences 13,14,22,23 suggest that RUX has been the principal driver of improved outcomes.…”

Section: Ruxmentioning

confidence: 68%

“…Despite the overall consensus that the survival of patients with MF has not changed significantly in the last several decades and remains poor, 9,10 there are recent reports suggesting a positive change, including our own experience 11 . Furthermore, there is significant emerging evidence that the survival improvement might be caused in part by the use of JAK inhibitors 12–15 …”

Section: Introductionmentioning

confidence: 98%

“…7 The third JAK inhibitor, pacritinib, received US Food and Drug Administration approval in February 2022 for patients with intermediate-risk or high-risk MF and platelets <50 � 10⁹/L using results from the phase 3 PERSIST-2 study (ClinicalTrials.gov identifier NCT02055781) 8 Despite the overall consensus that the survival of patients with MF has not changed significantly in the last several decades and remains poor, 9,10 there are recent reports suggesting a positive change, including our own experience. 11 Furthermore, there is significant emerging evidence that the survival improvement might be caused in part by the use of JAK inhibitors. [12][13][14][15] We designed this study with the specific goal of examining the survival of patients with MF from our institution based on therapy received.…”

Section: Introductionmentioning

confidence: 99%

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The role of therapy in the outcome of patients with myelofibrosis

Masárová

Bose

Pemmaraju

et al. 2023

Cancer

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BACKGROUNDThe treatment of patients with myelofibrosis (MF) has evolved in the past decade, as reflected in an increased use of various therapeutic agents that could potentially impact patient outcomes.METHODSIn this retrospective study, the authors evaluated the pattern of therapy and its possible impact on the survival of patients with MF at their institution. Patients (n = 802) with newly diagnosed, chronic, overt MF (MF fibrosis grade ≥2, <10% blasts) seen at their cancer center between 2000 and 2020 were included.RESULTSOverall, 492 of the included patients (61%) initiated MF‐directed therapy during follow‐up. The most frequent initial therapy was the JAK inhibitor ruxolitinib (44% of treated patients), investigational agents excluding JAK inhibitors (21%), immunomodulatory agents (18%), other investigational JAK inhibitors (10%), and others (7%). Overall survival was superior for patients who received initial ruxolitinib therapy, with a median survival of 72 months versus approximately 50 months for the remaining approaches, excluding the last group. Thirty‐two percent of patients required subsequent therapy (n = 159). The longest survival since the start of second‐line therapy was observed in patients who initiated salvage ruxolitinib (median, 35 months; 95% CI, 25–45 months).CONCLUSIONSThis study demonstrated improved outcomes of patients with MF who received treatment with the JAK inhibitor ruxolitinib.

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Section: Ruxmentioning

confidence: 68%

Section: Introductionmentioning

confidence: 98%

Section: Introductionmentioning

confidence: 99%

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The role of therapy in the outcome of patients with myelofibrosis

Masárová

Bose

Pemmaraju

et al. 2023

Cancer

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“… 1 The median overall survival (OS) in MF is ∼5 years. 2 However, prognosis is dismal in patients who progress to acute myeloid leukemia with an expected 98% mortality at a median of 2.5 months. 3 Leukemic transformation is reported in ∼14% of PMF, 4 8.2% of post-PV MF (PPV-MF), and 11.5% of post-ET MF (PET-MF).…”

Section: Introductionmentioning

confidence: 99%

JAK2 V617F mutation and associated chromosomal alterations in primary and secondary myelofibrosis and post-HCT outcomes

Rafati,

Brown,

Zhou

et al. 2023

Blood Advances

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JAK2 V617F is the most common driver mutation in primary or secondary myelofibrosis for which allogeneic hematopoietic cell transplantation (HCT) is the only curative treatment. Knowledge of the prognostic utility of JAK2 alterations in the HCT setting is limited. We identified all patients with MF who received HCT between 2000 and 2016 and had a pre-HCT blood sample (N = 973) available at the Center of International Blood and Marrow Transplant Research biorepository. PacBio sequencing and single nucleotide polymorphism–array genotyping were used to identify JAK2V617F mutation and associated mosaic chromosomal alterations (mCAs), respectively. Cox proportional hazard models were used for HCT outcome analyses. Genomic testing was complete for 924 patients with MF (634 primary MF [PMF], 135 postpolycythemia vera [PPV-MF], and 155 postessential thrombocytopenia [PET-MF]). JAK2V617F affected 562 patients (57.6% of PMF, 97% of PPV-MF, and 42.6% of PET-MF). Almost all patients with mCAs involving the JAK2 region (97.9%) were JAK2V617-positive. In PMF, JAK2V617F mutation status, allele burden, or identified mCAs were not associated with disease progression/relapse, nonrelapse mortality (NRM), or overall survival. Almost all PPV-MF were JAK2V617F-positive (97%), with no association between HCT outcomes and mutation allele burden or mCAs. In PET-MF, JAK2V617F high mutation allele burden (≥60%) was associated with excess risk of NRM, restricted to transplants received in the era of JAK inhibitors (2013-2016; hazard ratio = 7.65; 95% confidence interval = 2.10-27.82; P = .002). However, allele burden was not associated with post-HCT disease progression/relapse or survival. Our findings support the concept that HCT can mitigate the known negative effect of JAK2V617F in patients with MF, particularly for PMF and PPV-MF.

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“…Current pharmacotherapy of myelofibrosis in adults worldwide is based on two medicines authorized for use by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the Janus kinase inhibitors (JAKi), ruxolitinib and fedratinib [ 3 , 4 , 5 , 6 ], with recent studies indicating an increase in the median survival of patients in the last decade from 48 to 63 months [ 7 ]. Ruxolitinib is a Janus kinase inhibitor used to treat various types of myelofibrosis and polycythemia vera in patients who have not responded to or cannot tolerate hydroxyurea and to treat graft-versus-host disease in cases that are refractory to steroid treatment.…”

Section: Introductionmentioning

confidence: 99%

Study of Symptom Severity and Adherence to Therapy of Myelofibrosis Patients Treated with Ruxolitinib

et al. 2023

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We aimed to explore symptom severity and adherence to therapy for patients with myelofibrosis treated with ruxolitinib in Bulgaria. It is a prospective, non-interventional study performed at the specialized hospital for active treatment of hematological diseases in Sofia during 2022–2023. Date of diagnosis, demographic characteristics, clinical indicators, ruxolitinib dose, and other data points were collected. Clinical indicators were assessed at baseline, in the middle, and at the end of observation. Severity of symptoms was measured with MPN-SAF TSS and adherence to therapy with the Morisky 4 questionnaire six times during the observation. The mean age of diagnosis was 58.5 years, with the average duration of disease of 3 years. Patients’ laboratory results were within physiological ranges, with spleen size experiencing a constant decrease. The average value for the severity of the symptoms per MPN-SAF TSS results decreased significantly, indicating better disease control. The average adherence to therapy did not change and remained high at around 9 points, except for one patient. In conclusion the treatment of myelofibrosis patients with ruxolitinib decreased symptom severity and spleen size. Patients were adherent to the therapy over the observed period, but as treatment duration increases, the risk of adherence decreases.

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Improved survival of patients with myelofibrosis in the last decade: Single‐center experience

Cited by 23 publications

References 35 publications

The role of therapy in the outcome of patients with myelofibrosis

The role of therapy in the outcome of patients with myelofibrosis

JAK2 V617F mutation and associated chromosomal alterations in primary and secondary myelofibrosis and post-HCT outcomes

Study of Symptom Severity and Adherence to Therapy of Myelofibrosis Patients Treated with Ruxolitinib

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