Grafapex (treosulfan)

The use of non-treosulfan-based myeloablative conditioning (MAC) regimens increased the risk of endothelial complications compared to reduced-intensity conditioning (RIC) (HR, 4.9; 95% CI, 1.1-22.0; P=0.040), whereas outcomes with treosulfan-based MAC were comparable to RIC. In summary, allogeneic HSCT is a viable curative strategy for ED when performed before transformation to an aggressive malignancy i.e. myelodysplasia with excess blasts or acute myeloid leukemia. However, the elevated incidence of endothelial toxicity highlights the importance of optimizing conditioning intensity and enhancing peritransplant monitoring in this population.

P2, N=24, Not yet recruiting, Federal Research Institute of Pediatric Hematology, Oncology and Immunology

This is the first published case suggesting a potential role for leukotriene receptor antagonists in CYSLTR2-mutant UM. These findings support further preclinical and clinical investigation of montelukast as a repurposed therapy in this challenging disease entity.

P1, N=36, Recruiting, medac GmbH | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Aug 2025 --> Mar 2026

P2, N=82, Active, not recruiting, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

P2, N=27, Not yet recruiting, University Hospital Tuebingen

The multi-hit TP53 mutation was found to be significantly associated with a reduced LFS rate in MDS/AML patients, in comparison to those with a single-hit TP53 mutation. Patients with a single or multi-hit TP53 may achieve a long-term survival rate of approximately 30%, and should be considered as transplant candidates, even in the absence of complete remission.

P2, N=40, Active, not recruiting, Fred Hutchinson Cancer Center | Trial completion date: Dec 2026 --> Mar 2026

P2, N=23, Active, not recruiting, Fred Hutchinson Cancer Center | Recruiting --> Active, not recruiting | N=80 --> 23 | Trial completion date: Apr 2027 --> Jul 2026 | Trial primary completion date: Oct 2026 --> Jan 2026

Conditioning regimens were predominantly treosulfan-based (59%; with thiotepa, 34%); other patients received busulfan-based (24%) or melphalan-based (17%) regimens. This worldwide study expand data on HSCT for STAT3-HIES to 41 patients. Despite significant pre-HSCT pulmonary morbidity, OS was high, and patients have improved skin and respiratory disease, though the impact on extra-immune manifestations appears limited.

P2, N=220, Not yet recruiting, Technische Universität Dresden

Harnessing molecular technologies, clinical expertise, patient-centered decision-making, and innovative pharmaceutical strategies offers an exciting opportunity to shape a transformative and curative treatment framework. Here, we provide a contemporary review on HCT for MDS and MF, highlighting up-to-date insights into disease biology, standard of care, and recommendations, as well as open avenues.