busulfan

P1, N=48, Recruiting, Sawa Ito, MD | Not yet recruiting --> Recruiting

P2, N=120, Not yet recruiting, Peking University People's Hospital

An 8-year-old female patient achieved bone marrow remission with azacitidine and venetoclax and subsequently underwent allogeneic hematopoietic stem cell transplantation from an unrelated umbilical cord blood donor, The conditioning regimen included fludarabine, decitabine, busulfan, and cyclophosphamide, leading to sustained donor engraftment. At the 13-month follow-up, the patient remained in complete hematologic remission without recurrence. This case suggests that allo-HSCT is a feasible curative option for children with high-risk MDS and germline DDX41 predisposition, highlighting the critical importance of screening potential donors for the same mutation.

P3, N=340, Active, not recruiting, Merck Sharp & Dohme LLC | Recruiting --> Active, not recruiting

P=N/A, N=30, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China

P=N/A, N=82, Recruiting, Fujian Medical University Union Hospital | N=180 --> 82

P=N/A, N=31, Recruiting, Peking University People's Hospital | Phase classification: P4 --> P=N/A

P2, N=30, Completed, City of Hope Medical Center | Active, not recruiting --> Completed

P2, N=260, Active, not recruiting, City of Hope Medical Center | Trial completion date: Apr 2026 --> Mar 2027

Twenty-four newly diagnosed patients with GBM and unmethylated MGMT promoter were treated across eight cohorts following surgical resection and radiotherapy, testing Temferon doses ranging from 0.5 × 106 to 4.0 × 106 CD34+ cells kg-1 and different conditioning regimens (BCNU or busulfan, with or without thiotepa). Temferon is a safe and tolerable immunotherapeutic strategy in patients with newly diagnosed GBM. ClinicalTrials.gov: NCT03866109 .

Conditioning therapy included busulfan, thymoglobulin, and fludarabine, while graft-versus-host disease (GvHD) prophylaxis was administered with rituximab, methotrexate, and cyclosporine A. Nevertheless, he developed chronic cutaneous GVHD, which was treated for four years with ruxolitinib and tacrolimus, achieving only partial control...Surgery, radiation therapy, and immunotherapy with pembrolizumab led only to an initial partial response. This first pediatric case of immunotherapy for SCC in FA highlights the challenges of treating this rare patient group. Nevertheless, combining radiation therapy with immunotherapy may represent a possible option for disease control.

P=N/A, N=25, Not yet recruiting, Prof. Marc Ansari, Geneva University Hospitals