Key clinical point: Paroxysmal nocturnal hemoglobinuria (PNH) clones are highly prevalent in myelodysplastic syndrome (MDS). PNH positivity predicted higher response to immunosuppressive therapy (IST) and stem cell transplant s along with a favorable impact on overall survival (OS).
Major finding: PNH clones were present in 20.3% of MDS cases. PNH-positive vs. PNH-negative patients showed significantly higher response to IST (84% vs. 44.7%; P = .01) and stem cell transplant (71% vs. 56.6%; P = .09). PNH positivity had a favorable impact on disease progression ( P less than .01) and overall survival ( P less than .0001).
Study details : Data come from a large single-center study involving 3,085 patients with suspected underlying myeloid disorders, cytopenia, or unexplained thrombosis who underwent a first-time PNH test. The cohort had 869 cases of MDS.
Disclosures: The authors did not declare any source of funding. The authors declared no competing interests.
Source: Fattizzo B et al. Leukemia. 2021 Mar 4. doi: 10.1038/s41375-021-01190-9 .