Ruxolitinib in patients with graft versus host disease (GvHD): findings from a compassionate use program

The ruxolitinib compassionate use (CU) program offered ruxolitinib to patients ≥2 years of age with confirmed steroid-resistant acute or chronic graft-versus-host disease (aGvHD and cGvHD, respectively). Data from 1180 patients ( n = 775, 370 and 35 with cGvHD, aGvHD, and non-specified GvHD, respec...

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Veröffentlicht in:	Bone marrow transplantation (Basingstoke) 2024-05, Vol.59 (5), p.637-646
Hauptverfasser:	Pattipaka, Thirupathi, Sarp, Séverine, Nakhaei, Peyman, Güneş, Sibel
Format:	Artikel
Sprache:	eng
Schlagworte:	692/699/249/1529 692/700/565/1436/2185 Adolescent Adult Aged Cell Biology Child Child, Preschool Compassionate Use Trials Corticoids Corticosteroids Female Graft versus host disease Graft vs Host Disease - drug therapy Graft-versus-host reaction Hematology Hematopoietic Stem Cell Transplantation Humans Inhibitor drugs Internal Medicine Male Medicine Medicine & Public Health Middle Aged Nitriles Public Health Pyrazoles - therapeutic use Pyrimidines - therapeutic use Stem Cells Steroids Young Adult
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Zusammenfassung:	The ruxolitinib compassionate use (CU) program offered ruxolitinib to patients ≥2 years of age with confirmed steroid-resistant acute or chronic graft-versus-host disease (aGvHD and cGvHD, respectively). Data from 1180 patients ( n = 775, 370 and 35 with cGvHD, aGvHD, and non-specified GvHD, respectively) were analyzed. Most patients had severe cGvHD (56%) or stage III/IV aGvHD (70%) disease and had previously received corticosteroids ( > 80%); ruxolitinib was requested primarily as a second-/third-line option. Patients
ISSN:	0268-3369 1476-5365 1476-5365
DOI:	10.1038/s41409-024-02207-4