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 3 publications

(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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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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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Efficacy of Ruxolitinib with corticosteroids in idiopathic pneumonia syndrome post-allogeneic hematopoietic stem cell transplantation: A single-center experience and systematic review

Shahzad,

Khan,

Amin

et al. 2024

Transplant Immunology

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Two machine learning-derived nomogram for predicting the occurrence and severity of acute graft-versus-host disease: a retrospective study based on serum biomarkers

He,

Li,

Fang

et al. 2024

Front. Genet.

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BackgroundAcute graft-versus-host disease (aGVHD) is a common complication after allogeneic hematopoietic cell transplantation (allo-HSCT), with high morbidity and mortality. Although glucocorticoids are the standard treatment, only half of patients achieve complete remission. Thus, there is an urgent need to screen biomarkers for the diagnosis of aGVHD to assist in the identification of individuals at risk of aGVHD. This study was to construct prediction models for the occurrence and severity of aGVHD using two machine learning algorithms based on serum biochemical data.MethodsClinical data of 120 patients with hematological diseases who received allo-HSCT were retrospectively analyzed. Seventy-six patients developed aGVHD, including 56 grade I/II and 20 grade III/IV. First, 15 serum biochemical indicators were considered as potential risk factors, and the differences in the levels of indicators between non-aGVHD and aGVHD were observed, followed by evaluation of the diagnostic property. Subsequently, to develop the prediction models for the occurrence and severity of aGVHD, LASSO and random forest (RF) analyses were performed with experimental indicators. Finally, Venn diagram analysis was utilized to obtain shared biomarkers in the two algorithms to construct the nomogram. The model performance was measured by calibration curves. Internal and external validations were performed based on risk score models and ROC curve analyses.ResultsTotal 12 of 15 indicators exhibited significant differences between the aGVHD and non-aGVHD groups, with AUC values > 0.75. In machine learning analysis, eight features (LAG-3, TLR-2, PD-L1, IP-10, elafin, REG-3α, ST2, TIM3) and seven variables (LAG-3, TLR-2, PD-1, Flt_3, IL-9, elafin, TIM3) were selected to distinguish aGVHD vs. non-aGVHD as well as grade I/II vs. III/IV, respectively. Further, the corresponding nomogram models were established and calibration curves showed that prediction was in good agreement with the actual probability. Biomarker-based risk score model was constructed, which obtained AUC value >0.89 in internal and external datasets.ConclusionClinical variables screened through learning algorithm can predict the risk and severity of aGVHD. Our findings may help clinicians develop more personalized and reasonable management strategies.

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

Cited by 3 publications

References 98 publications

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

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

Efficacy of Ruxolitinib with corticosteroids in idiopathic pneumonia syndrome post-allogeneic hematopoietic stem cell transplantation: A single-center experience and systematic review

Two machine learning-derived nomogram for predicting the occurrence and severity of acute graft-versus-host disease: a retrospective study based on serum biomarkers

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