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Risk of mortality and second malignancies in primary myelofibrosis before and after ruxolitinib approval
John W Thomas1, Omer Jamy2, Mithun Vinod Shah3
1Tinsley Harrison Internal Medicine Residency Program, University of Alabama at Birmingham (UAB), Birmingham, AL, USA.
Background:
Primary myelofibrosis (PMF) is associated with morbidity and mortality. Ruxolitinib gained US FDA approval for treatment of intermediate/high-risk PMF in November 2011. We evaluated differences in survival and second primary malignancy (SPM) incidence among US PMF patients in the years before and after ruxolitinib approval.
Methods:
We conducted a retrospective study utilizing the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER)-18 database for PMF patients. We divided patients into five-year cohorts pre- (2007-2011) and post-ruxolitinib (2012-2016) approval and compared relative survival rates (RSRs) to the standard population and standardized incidence rates (SIRs) of SPMs between cohorts.
Results:
We included 2020 patients diagnosed with PMF from 2007-2016 in this study. There was no difference in the four-year RSRs between cohorts (54 % vs. 57 %, p = 0.776). More patients developed SPMs in the post-ruxolitinib cohort (8% vs. 6%, p = 0.041). The majority of SPMs were hematologic with higher incidence of AML transformation in the post-ruxolitinib cohort (SIR 125.29 vs. 70.55).
Conclusions:
PMF prognosis remains poor in the years following ruxolitinib's approval. SPM incidence including AML transformation is higher in the years after approval. Further studies are needed to determine the true impact of ruxolitnib on population outcomes.
Insights
Survival for primary myelofibrosis (PMF) patients did not change after ruxolitinib approval. However, the incidence of second primary malignancies (SPMs), including AML transformation, increased post-approval.
Area of Science:
- Hematology
- Oncology
- Epidemiology
Background:
- Primary myelofibrosis (PMF) is a serious condition with significant morbidity and mortality.
- Ruxolitinib was approved by the US FDA in November 2011 for treating intermediate/high-risk PMF.
Purpose of the Study:
- To evaluate survival differences in US PMF patients before and after ruxolitinib approval.
- To assess changes in second primary malignancy (SPM) incidence following ruxolitinib's introduction.
Main Methods:
- Retrospective study using the National Cancer Institute's SEER-18 database.
- Comparison of five-year cohorts: pre-ruxolitinib (2007-2011) and post-ruxolitinib (2012-2016).
- Analysis included relative survival rates (RSRs) and standardized incidence rates (SIRs) of SPMs.
Main Results:
- No significant difference in four-year RSRs between the pre- and post-ruxolitinib cohorts (54% vs. 57%, p=0.776).
- Increased incidence of SPMs in the post-ruxolitinib cohort (8% vs. 6%, p=0.041).
- Higher incidence of hematologic SPMs, particularly AML transformation, was observed post-approval (SIR 125.29 vs. 70.55).
Conclusions:
- PMF prognosis remains poor despite ruxolitinib approval.
- Increased SPM incidence, including AML transformation, noted in the post-ruxolitinib era.
- Further research is necessary to ascertain ruxolitinib's overall population impact.
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