Gagelmann N, Badbaran A, Salit RB, et al. Impact of TP53 on Outcome of Patients with Myelofibrosis Undergoing Hematopoietic Stem Cell Transplantation. Blood. 2023; (doi: 10.1182/blood.2023019630).

For patients with myelofibrosis undergoing hematopoietic stem cell transplantation, multi-hit TP53 mutations (TP53^MT) are a very high risk group, while the outcomes of those developing single-hit TP53^MT were similar to those without mutations, new research shows. The study included 349 patients, with TP53^MT detected in 49, including 30 who had a multi-hit configuration. The median variant allele frequency was 20.3%, while cytogenic risk was favorable for 71%, unfavorable for 23%, and very high for 6%. Thirty-six patients (10%) had complex karyotypes. TP53^MT patients had a 1.5-year median survival rate compared with 13.5 years for patients with TP53 wild-type (TP53^WT). Six-year survival for multi-hit TP53^MT patients was 25%, compared with 56% for single-hit TP53^MT patients and 64% for TP53^WT carriers. Transplant-specific risk factors and conditioning intensity did not affect outcomes. Single-hit TP53^MT patients had a 17% cumulative incidence of relapse vs 52% for multi-hit TP53^MT patients and 21% for TP53^WT. Ten TP53^MT patients had leukemic transformation soon after transplantation compared with seven TP53^WT patients. Eight of 10 patients with TP53^MT had a multi-hit configuration. The median time to leukemic transformation for multi-hit TP53^MT, single-hit TP53^MT, and TP53^WT was 0.7 years, 0.5 years, and 2.5 years, respectively.

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Tags: hematology, Research, Science, abstract, mutation, results, hematopoietic transplantation