Ruxolitinib in Acute and Chronic Graft-Versus-Host Disease: Real Life Experience in a Multi-Centre Study

Gomez, Virginia Escamilla; Gutiérrez, Valentín García; Mahillo, Beatriz Astibia; Alcalde, Patricia; Corral, Lucía López; Gómez, Marina Acera; Torres, Melissa; Borrego, Asunción Borrego; Pinedo, Leslie González; Zudaire, Maite; Vicent, Marta González; Benzaquén, Ana; Garcia, Isabel Izquierdo; Cantó, Pedro Asensi; Montoro, Juan; Pascual, Guillermo Orti; Valcárcel, David; Benítez‐Carabante, María Isabel; Rubio, C. Díaz de Heredia; Giro, Eloi Cañamero; Ferrà, Christelle; García‐Cadenas, Irene; Redondo, Sara; Sisinni, Luisa; Pérez, Antonio; Mussetti, Alberto; García, Lucia; Moraleda, María Del Pilar Palomo; Sierra, Pedro Antonio González; Chacón, Manuel; Pérez‐Simón, José A.

doi:10.1182/blood-2022-164561

Cited by 3 publications

(8 citation statements)

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“…As expected, the patient population in this global ruxolitinib CU program was heterogenous and heavily pretreated, including pediatric patients <12 years old, predominantly with cGvHD and often with severe disease, which is consistent with results from other real-world studies with ruxolitinib [ 29 , 30 ] and a US registry [ 31 ]. The REACH studies also investigated patients with late stage and severe disease (64% stage III–IV aGvHD; 57% had severe cGvHD) [ 14 , 15 ] but the inclusion of pediatric patients (from 2 years old) and the number of patients treated in this CU program, and in other ruxolitinib real-world studies [ 29 – 31 ] have greatly expanded the ruxolitinib-treated population beyond the REACH trial populations ( ≥ 12 years old) [ 14 , 15 ].…”

Section: Discussionsupporting

confidence: 87%

“…The REACH studies also investigated patients with late stage and severe disease (64% stage III–IV aGvHD; 57% had severe cGvHD) [ 14 , 15 ] but the inclusion of pediatric patients (from 2 years old) and the number of patients treated in this CU program, and in other ruxolitinib real-world studies [ 29 – 31 ] have greatly expanded the ruxolitinib-treated population beyond the REACH trial populations ( ≥ 12 years old) [ 14 , 15 ]. Consistent with other CU programs [ 28 – 30 , 32 ] many patients in this study had received multiple lines of therapy and concomitant medications, which highlights the complexity of their disease. Interestingly, ruxolitinib was considered predominantly as a second- or third-line treatment option, which suggests that physicians’ have confidence in the efficacy of ruxolitinib versus investigators’ choice of treatment as demonstrated in patients with steroid-refractory aGvHD and cGvHD in the REACH studies, and its safety in vulnerable patients [ 14 , 15 ].…”

Section: Discussionsupporting

confidence: 68%

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

Pattipaka,

Sarp,

Nakhaei

et al. 2024

Bone Marrow Transplant

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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 <12 and ≥12 years old most often received the recommended ruxolitinib doses (5 mg twice daily [BID] and 10 mg BID, respectively); however, 23% and 30% of ≥12 year olds with cGvHD and aGvHD, respectively, received the lower dose of 5 mg BID. Notably, corticosteroid usage decreased with ruxolitinib treatment; at the initial ruxolitinib request, 81% and 91% of patients with cGvHD and aGvHD, respectively, were receiving corticosteroids whereas at resupply, 62% and 64%, respectively, were receiving corticosteroids. Eighty two percent of evaluable patients with cGvHD had a complete or partial response to treatment and 56% of evaluable patients with aGvHD had a best response of grade 0/I. These findings demonstrate the rapid and positive effects of ruxolitinib in patients with GvHD in a real-world setting.

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Section: Discussionsupporting

confidence: 87%

Section: Discussionsupporting

confidence: 68%

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

Pattipaka,

Sarp,

Nakhaei

et al. 2024

Bone Marrow Transplant

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“…Previous studies showed that the ORR of ruxolitinib in refractory cGVHD treatment was approximately 43.5% to 100%, and the CRR was from 3.5% to 13.0%, with a median time to best response ranging from 2 to 4 weeks. 8,[12][13][14] In the present study, the ORR was 70.7% (95% CI, 56.2%-85.3%), and the CRR was 36.6% (95% CI, 21.2%-52.0%). The median time to reach the best response was 2.0 months.…”

Section: Discussionsupporting

confidence: 46%

“…The statistical results showed an association between lung cGVHD and treatment response, which was also reported in a previous study. 13 The multivariate analysis indicated that among patients with cGVHD, lung involvement was associated with a higher risk of treatment failure.…”

Section: Discussionmentioning

confidence: 96%

Evaluation of Ruxolitinib for Steroid-Refractory Chronic Graft-vs-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Shi

Luo

et al. 2021

JAMA Netw Open

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Key Points Question Is ruxolitinib an option for patients with steroid-refractory chronic graft-vs-host disease, and what characteristics are associated with treatment response? Findings In this case series of 41 patients with steroid-refractory chronic graft-vs-host disease who were treated with ruxolitinib, heavily pretreated patients could achieve meaningful responses with a favorable safety profile. No lung involvement and haploidentical donors were associated with response to ruxolitinib. Meaning In this study, monotherapy with ruxolitinib was associated with a meaningful response in patients with steroid-refractory chronic graft-vs-host disease, suggesting a possible therapeutic option for a serious disease with no currently accepted standard-of-care treatment.

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“…8,14,15 Several groups also report fungal infections, most commonly pulmonary aspergillosis, associated with ruxolitinib use for GVHD. 11,14,17,18 One case of CMV reactivation occurred in our cohort, but no cases of invasive fungal infection or aspergillosis occurred in our patients. Most of our patients with active cGVHD were receiving antifungal prophylaxis with aspergillus activity while on ruxolitinib.…”

Section: Discussionmentioning

confidence: 52%

Ruxolitinib for the Treatment of Chronic GVHD and Overlap Syndrome in Children and Young Adults

et al. 2022

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Ruxolitinib in Acute and Chronic Graft-Versus-Host Disease: Real Life Experience in a Multi-Centre Study

Cited by 3 publications

References 0 publications

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

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

Evaluation of Ruxolitinib for Steroid-Refractory Chronic Graft-vs-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Ruxolitinib for the Treatment of Chronic GVHD and Overlap Syndrome in Children and Young Adults

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