Researchers have developed a myelofibrosis transplant scoring system (MTSS) that can be predictive of allogeneic hematopoietic stem cell transplantation (HCT) outcomes for patients with primary and post-essential thrombocythemia/polycythemia vera (ET/PV) myelofibrosis. The MTSS can help determine patient eligibility for HCT based on HCT-specific risk stratification.
Researchers collected clinical, molecular and transplant-specific information from 361 patients with myelofibrosis. From the total cohort, 205 patients made up the training cohort to create the MTSS tool. The remaining 156 patients provided external validation of the tool.
Independent prognostic factors determined to be associated with HCT survival based on multivariable analysis included:
- Age ≥ 57 years
- Karnofsky performance status < 90%
- Platelet count < 150 x 109/L and leukocyte count > 25 x 109/L prior to HCT
- HLA-mismatched unrelated donor
- ASXL1 mutation and non-CALR/MPL driver mutation genotype
Researchers used these risk factors to develop the MTSS’s four levels of risk stratification ranging from low to very high, with five-year survival by risk group: 83% (95% CI, 71-95%); 64% (95% CI, 53-75%); 37% (95% CI, 17-57%); and 22% (95% CI, 4-39%), respectively. An increasing score was predictive of non-relapse mortality. When compared to currently available disease-specific systems, the MTSS improved the ability to predict survival (p<0.001) after HCT.
The researchers concluded that, when used with disease-specific systems, the scoring system may help with clinical decision making for patients who are eligible for HCT and counseling patients about potential outcomes post-HCT.