Tyrosine kinase inhibitors (TKIs) approved for chronic myeloid leukemia known to have similar efficacies but different safety profiles. Therefore, the choice of patient-specific treatments is driven by factors such as tolerability and adverse event profile of TKIs. This review article examines the most up-to-date data and provides practical recommendations for clinical approaches. Nilotinib and ponatinib should be avoided in patients with cardiovascular risk factors, dasatinib in patients with lung damage and bosutinib and nilotinib in patients with liver disease. Considering that certain comorbidities predispose some patients to developing severe adverse events when receiving TKIs, the first- and second-line treatment of chronic myeloid leukemia should be tailored to each patient’s individual condition.

Although inhibition of complement system at different steps is a promising therapy modality in PNH (paroxysmal paroxysmal nocturnal hemoglobinuria) patients, allogeneic hematopoetic stem cell transplantation (HCT) is still the only curative therapy especially for patients with intractable hemolysis or bone marrow failure. The aim of this study is to evaluate the outcomes of allogeneic HCT in PNH patients with or without aplastic anemia ( PNH-AA). 35 PNH / PNH-AA patients who were treated with allegeneic HCT in ten transplantation centers in Turkey were retrospectively analyzed. 16 (45.7 %) and 19 (54.3 %) patients were diagnosed as classical PNH and PNH / AA respectively. The median age of the patients was 32 ( 18-51) 2-year overall survival (OS) and GVHD-free, failure-free survival (GFFS) was 81.2 % and 78.1 % , respectively. 2 year OS in classical PNH and PNH /AA was 81.3 % and % 79.9 (p =0,87), respectively and 2 year GFFS in PNH and PNH/AA was 79% and 76% (0,977) without statistical significance. OS and GFFS rates did not differ between transplantations with matched sibling donor (MSD) and matched unrelated donor (MUD), neither. Allo HCT with MSD or MUD is a good option in selected patients with classical PNH and PNH / AA. Especially, patients with debilitating and refractory hemolysis and patients with bone marrow failure might form the excellent group for allo - HCT.

This retrospective study evaluated 35 children (median age 5.2 years; range 0.4–18) with myelofibrosis (MF), including 33 with primary myelofibrosis and 2 with secondary myelofibrosis transplanted from matched sibling donor (MSD) (n = 17) or non-MSD (n = 18) between 2000 and 2022. Conditioning was usually chemotherapy-based (n = 33) and myeloablative (n = 32). Fifteen patients received bone marrow (BM), 14 haematopoietic cells (HC) from peripheral blood (PB), and 6 from cord blood (CB). Day +100 acute GvHD II–IV incidence was significantly lower after MSD-haematopoietic cell transplantation (MSD-HCT) than after non-MSD-HCT [18.8% (4.3–41.1) vs 58.8% (31–78.6); p = 0.01]. Six-year non-relapse mortality (NRM) was 18% (7.1–32.8), relapse incidence was 15.9% (5.6–30.9), progression-free survival (PFS) was 66.1% (47–79.7), GvHD-free relapse-free survival was 50% (30.6–66.7), and overall survival (OS) was 71.1% (51.4–84). Six-year PFS and OS were significantly higher after BM transplantation compared to HCT from other sources [85.1% (52.3–96.1) vs 50.8% (26.3–71), p = 0.03, and 90.9% (50.8–98.7) vs 54% (28.1–74.2), p = 0.01, respectively], whereas NRM was significantly lower [0% vs 32% (12.3–53.9); p = 0.02]. This first multicentre study on outcomes of allogeneic HCT in children with myelofibrosis proves feasibility and curative effect of transplantation in these children, suggests that bone marrow transplantation is associated with better outcomes, and indicates the need for further studies.