melphalan

P=N/A, N=13, Terminated, Institut Cancerologie de l'Ouest | Phase classification: P1/2 --> P=N/A

dexamethasone, ara-C, cisplatin (DHAP) was the most common salvage regimen (47.8%)...BCNU, etoposide, ara-C, melphalan (BEAM) was the most common conditioning regimen 91.9%...t-MN significantly contributes to NRM post-auto-HCT. Age at transplant is the primary risk factor, highlighting the need for vigilant monitoring and risk mitigation strategies.

However, to our best knowledge, there has been no report about modification of fludarabine (Flu)/melphalan (Mel)-based PTCy-haplo in a real-world setting...In addition, despite the poor baseline characteristics including older age (median, 59 years [range, 25-72]), active diseases at the time of PTCy-haplo (n=8), and short interval between the first and second allogeneic hematopoietic stem cell transplantation (allo-HSCT) (n=8; median, 12.4 months [range, 3.9-32.9]; all patients received Flu/busulfan (Bu)-based conditioning regimen for their first allo-HSCT), only two patients (10.5%) developed sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD). With a median follow-up of 30.2 months (range, 1.0-53.1), overall survival and disease-free at 2 years were 56.8 and 51.5%, respectively. These findings suggested that early TAC initiation using Flu/Mel-based PTCy-haplo may be potentially useful for older patients with low tolerance to CRS, high risk of SOS/VOD or recurrence, or for those who are unlikely to receive Bu-based conditioning regimen due to early relapse after allo-HSCT with Bu-based conditioning regimen.

In the randomized SCANDIUM II trial, patients with metastatic UM received a one-time treatment with isolated hepatic perfusion using high-dose melphalan in combination with systemic immune checkpoint inhibition with ipilimumab (3 mg/kg) and nivolumab (1 mg/kg). Patients with undetectable ctDNA 2-4 months after the start of treatment had significantly improved progression-free survival (P = 0.024), and a non-significant improvement in overall survival. In patients with UM liver metastases treated with combined hepatic perfusion and immune checkpoint inhibition, ctDNA may serve as a predictive biomarker and warrants further validation.

P2, N=32, Completed, University Hospital, Lille | Unknown status --> Completed | N=80 --> 32

P2, N=31, Active, not recruiting, City of Hope Medical Center | Trial completion date: Nov 2025 --> Jul 2026 | Trial primary completion date: Nov 2025 --> Jul 2026

P1, N=28, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Nov 2025 --> Nov 2026 | Trial primary completion date: Nov 2025 --> Nov 2026

P2, N=33, Recruiting, City of Hope Medical Center | Trial completion date: Oct 2027 --> Oct 2029 | Trial primary completion date: Oct 2027 --> Oct 2029

P4, N=450, Active, not recruiting, University of Turin, Italy | Recruiting --> Active, not recruiting

These results demonstrate that model-based ATG dosing promotes robust CD4+ immune reconstitution after ex vivo CD34+ selected allogeneic HCT, underscoring the potential of pharmacokinetically guided ATG as a strategy to optimise immune recovery in myeloablative, calcineurin inhibitor-free transplantation for haematological malignancies.

MCM6 acts as a critical regulator of retinoblastoma growth and modulates response to melphalan. Targeting MCM6 may offer a therapeutic approach to improve outcomes of chemotherapy in retinoblastoma.

P2, N=40, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China