Ixazomib in the Prophylaxis of Chronic Graft-versus-host Disease.

Summary

Allogeneic hematopoietic stem cell transplantation (HTC) is the only curative option for many patients with hematologic malignancies but \>50% of this patients will develop extensive chronic graft-versus-host disease (cGVHD), which remains the most important complication after HTC. Classically, the most effective strategies to prevent GVHD have not improved survival; therefore, the new strategies are being sought. This study is designed in two phases: the main objective for phase I study is the more suitable dose for ixazomib search. Phase II study is designed to evaluate the efficacy of ixazomib at the doses stablished in phase I.

Timeline

Start

2017-05-16

Primary completion

2024-01-11

Completion

2024-12-31

Outcome

Met primary endpoint

paper the cumulative incidence of moderate/severe cGVHD in the IXZ vs control groups at 1 and 2 years were: 3.23% vs 30.2% (hazard ratio [HR], 0.089; P = .02) PMID 40737543 ↗

paper The administration of IXZ decreases the risk of moderate/severe cGVHD. PMID 40737543 ↗

Publications

• Caballero-Velazquez T, Delgado Serrano J, Lopez-Corral L, Ferra-Coll C, Garcia-Calderon CB, Valcarcel D, Garcia-Cadenas I, Perez Lopez E, Jimenez Lorenzo MJ, Martin-Dominguez FM, Jimenez-Leon MLR, Orti G, Escamilla Gomez V, Blazquez-Goni C, Cabero Martinez A, Andrade Ruiz H, Menendez-Pedregal E, Sanchez-Guijo F, Perez Simon JA. Ixazomib decreases the risk of chronic graft-versus-host disease: identification of cGVHD biomarkers. Blood Adv. 2025 Nov 11;9(21):5528-5538. doi: 10.1182/bloodadvances.2025016284.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ixazomib	Unknown	3 mg	Oral
Subject	Ixazomib	Unknown	4 mg	Oral
Background	Sirolimus	Small molecule	4 mg	Oral
Background	Tacrolimus	Small molecule	0.06 mg/kg	—

Indications