Ruxolitinib as the first post-steroid treatment for acute and chronic graft-versus-host disease

Algeri, Mattia; Becilli, Marco; Locatelli, Franco

doi:10.1080/1744666x.2023.2249230

Cited by 1 publication

(2 citation statements)

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“…Use of non-conventional treatments as ECP and MSC resulted in good response rates (from 66 to 80% in principal studies) but less feasibility due to technical requirements [ 59 – 61 ]. In this regards, ruxolitinib brings together efficacy, manageability, and lower immunosuppressive impact even in acute patients, compared to other treatment options [ 2 , 62 , 63 ].…”

Section: Discussionmentioning

confidence: 99%

“…It is important to note that pediatric cGvHD represents a multi-system disease with variable individual clinical course. In this view, a personalized approach needs to be adopted, highlighting the importance of early diagnosis and treatment and taking into consideration the clinical status and individual needs of each patient when choosing the most appropriate treatment option [ 62 , 63 , 67 ].…”

Section: Discussionmentioning

confidence: 99%

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Ruxolitinib for the treatment of acute and chronic graft-versus-host disease in children: a systematic review and individual patient data meta-analysis

Baccelli,

Gottardi,

Muratore

et al. 2024

Bone Marrow Transplant

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Steroid-refractory graft-versus-host disease (SR-GvHD) represents a major complication of pediatric allogenic hematopoietic stem cell transplantation. Ruxolitinib, a selective JAK 1–2 inhibitor, showed promising results in the treatment of SR-GvHD in adult trial, including patients >12 years old. This systematic review aims to evaluate ruxolitinib use for SR-GvHD in the pediatric population. Among the 12 studies included, ruxolitinib administration presented slight differences. Overall response rate (ORR) ranged from 45% to 100% in both acute and chronic GvHD. Complete response rates (CR) varied from 9% to 67% and from 0% to 28% in aGvHD and cGvHD, respectively. Individual-patient meta-analysis from 108 children under 12 years showed an ORR and CR for aGvHD of 74% and 56%, respectively, while in cGvHD ORR was 78% but with only 11% achieving CR. Treatment-related toxicities were observed in 20% of patients, including cytopenia, liver toxicity, and infections. Age, weight, graft source, previous lines of therapy, and dose did not significantly predict response, while a higher rate of toxicities was observed in aGvHD patients. In conclusion, ruxolitinib shows promising results in the treatment of SR-GvHD in children, including those under 12 years. Specific pediatric perspective trials are currently ongoing to definitely assess its efficacy and safety.

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