Contemporary risk models for patients with primary myelofibrosis aim to identify patients for whom early transplant is appropriate. A study published in the American Journal of Hematology offered a phenotypic profile that might predict median survival of 20 years or more in patients with primary myelofibrosis, thereby allowing more informed risk assessment for early transplant.
Researchers retrospectively reviewed the medical records of 1282 patients with primary myelofibrosis for variables including the presence of constitutional symptoms (significant weight loss, unexplained sweats, or fever lasting at least 1 month), anemia, various genetic mutations, and number of platelets, leukocytes, and circulating blasts.
Constitutional symptoms were detected in 29% of patients; high molecular risk mutations were present in 61% of patients. Leukocytosis, thrombocytopenia, and 2% or higher circulating blasts had incidence rates of 14%, 23%, and 28%, respectively. Anemia was present in 55% of patients.
In total, 626 patients died within 5 years of diagnosis and 26 patients survived for at least 20 years. The latter group was found to be younger, more likely to be female, and less likely to have constitutional symptoms, anemia, leukocytosis, thrombocytopenia, or 2% or higher circulating blasts. Patients who survived for 20 years or more were also less likely to have unfavorable or very high risk karyotypes as determined by a cytogenetics risk model. Conversely, presence of anemia, leukocytosis, thrombocytopenia, or 2% or higher circulating blasts were found to be independently predictive of death within 5 years of diagnosis.
Of note, presence of high molecular risk mutations was not associated with odds for long-term survival, and presence of palpable splenomegaly was not associated with odds for short-term or long-term survival.
The authors concluded that their results “complement conventional risk models in primary myelofibrosis” and “should facilitate treatment decision-making for the individual patient.”
1. Penna D, Lasho TL, Finke CM, et al. 20+ years and alive with primary myelofibrosis: phenotypic signature of very long-lived patients [published online December 5, 2018]. Am J Hematol. doi: 10.1002/ajh.25351