“MDS/MPN with neutrophilia is a very rare disease. It carries a poor median overall survival of 15 months (with a reported range of 12.4 to 37 months),” the study authors explained in their report. “Without treatment, 30-40% of patients progress to acute myeloid leukemia.”
Researchers retrospectively analyzed a cohort of 347 adult patients diagnosed with MDS/MPN with neutrophilia, previously known as atypical chronic myeloid leukemia, who were registered in the Netherlands Cancer Registry between 2001 and 2019. The aim of the study was to validate known prognostic markers, identify novel prognostic markers, and provide evidence-based treatment recommendations.
The demographic baseline data of the cohort was consistent with those of cohorts from other studies. Most patients were male (65%) and >65 years old (71%). The median age at diagnosis was 72 years (range, 22-95 years). Only 5 patients (1.4%) were known to have a prior hematological malignancy (MDS, n=2; lymphoma, n=3). The median OS for the overall cohort was 15.8 months (95% CI, 13.8-17.2 months), and no significant difference in OS was observed between the sexes.
Among 110 patients diagnosed between 2014-2019, cytogenetic testing data was available for 89% of patients. Of those, 15% had cytogenetic abnormalities, which were all in patients aged >65 years. The most common cytogenetic abnormality was trisomy 8 (6/15 patients).
Within the same subgroup, molecular analysis was available for 92% of patients. Of those, 49 patient harbored a total of 16 distinct molecular mutations, with some patients (16/101) having up to 3 different mutations. The most frequent mutations were in ASXL1 (22%), SETBP1 (18%), SRSF2 (12%), and CSFR3 (12%).
In the overall cohort, a multivariable Cox regression analysis adjusted for primary therapy revealed that age (>65 years of age hazard ratio [HR], 1.85; 95% CI, 1.34-2.55; P =.001) and alloHSCT (HR, 0.51; 95% CI, 0.26-0.97; P =.039) were associated with OS.
“As no other treatment modality, seemed to impact survival and might cause toxicity, we propose that all patients eligible for alloHSCT should whenever possible receive an allogeneic transplant. It is imperative that we strive to improve outcomes for patients not eligible for alloHSCT,” the study authors concluded in their report.
Reference
Klein SK, Huls GA, Visser O, Kluin-Nelemans HC, Dinmohamed AG. Characteristics, primary treatment, and survival of MDS/MPN with neutrophilia: a population-based study. Blood Adv. Published online November 7, 2023. doi:10.1182/bloodadvances.2023011181