Budget impact analysis of belumosudil for chronic graft-versus-host disease treatment in the United States

Bachier, Carlos; Skaar, Jeffrey R.; Dehipawala, Sumudu; Miao, Benjamin; Ieyoub, Jonathan; Taitel, Haya

doi:10.1080/13696998.2022.2087408

Cited by 3 publications

(1 citation statement)

References 16 publications

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“…The best response rate for belumosudil at 200 mg twice daily was 77%, and symptom reduction was reported in 62% of patients ( 82 ). Belumosudil taken once daily was reported in a 2022 article to cost around $232,000 per year ( 83 ). In September 2021, approval of ruxolitinib was expanded to include treatment for cGVHD in adult and pediatric patients ages twelve and older after failure of ≤2 lines of systemic therapy with almost 50% ORR and median failure-free survival of >18 months demonstrated in the REACH3 Phase 3 study ( 16 , 84 ).…”

Section: Graft Versus Host Disease Characteristics and Manifestationsmentioning

confidence: 99%

Novel therapies for graft versus host disease with a focus on cell therapies

Zeiser,

Ringden,

Sadeghi

et al. 2023

Front. Immunol.

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Graft versus host disease (GVHD) can occur at any period post allogeneic hematopoietic stem cell transplantation as a common clinical complication contributing to significant morbidity and mortality. Acute GVHD develops in approximately 30-50% of patients receiving transplants from matched related donors. High doses of steroids are used as first-line treatment, but are unsuccessful in around 40% of patients, resulting in the diagnosis of steroid-refractory acute GVHD. Consensus has yet to develop for the management of steroid-refractory acute GVHD, and prognosis at six months has been estimated at around 50%. Thus, it is critical to find effective treatments that increase survival of steroid-refractory acute GVHD. This article describes the currently known characteristics, pathophysiology, and treatments for GVHD, with a special focus on recent advances in cell therapies. In particular, a novel cell therapy using decidua stromal cells (DSCs) was recently shown to have promising results for acute GVHD, with improved effectiveness over previous treatments including mesenchymal stromal cells. At the Karolinska Institute, severe acute GVHD patients treated with placenta-derived DSCs supplemented with either 5% albumin or 10% AB plasma displayed a one-year survival rate of 76% and 47% respectively. Furthermore, patients with steroid-refractory acute GVHD, displayed survival rates of 73% with albumin and 31% with AB plasma-supplemented DSCs, compared to the 20% survival rate in the mesenchymal stromal cell control group. Adverse events and deaths were found to be attributed only to complications of hematopoietic stem cell transplant and GVHD, not to the study intervention. ASC Therapeutics, Inc, in collaboration with the Karolinska Institute, will soon initiate a phase 2 multicenter, open-label study to further assess the efficacy and safety of intravenous DSC treatment in sixty patients with Grade II-IV steroid-refractory acute GVHD. This novel cell therapy represents a promising treatment to combat the poor prognosis that steroid-refractory acute GVHD patients currently face.

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Section: Graft Versus Host Disease Characteristics and Manifestationsmentioning

confidence: 99%

Novel therapies for graft versus host disease with a focus on cell therapies

Zeiser,

Ringden,

Sadeghi

et al. 2023

Front. Immunol.

View full text Add to dashboard Cite

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Navigating the Complexity of Chronic Graft-vs-Host Disease: Canadian Insights into Real-World Treatment Sequencing

Kim,

Taparia,

Robinson

et al. 2024

Transplantation Proceedings

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Treatment of steroid-refractory graft versus host disease in children

Gottardi,

Leardini,

Muratore

et al. 2023

Front. Transplant.

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Systemic steroids are still the first-line approach in acute graft-versus-host disease (aGvHD), and the backbone of chronic GvHD management. Refractoriness to steroid represent a major cause of morbidity and non-relapse mortality after hematopoietic stem cell transplantation (HSCT). In both backgrounds, several second-line immunosuppressive agents have been tested with variable results in terms of efficacy and toxicity. Solid evidence regarding these approaches is still lacking in the pediatric setting where results are mainly derived from adult experiences. Furthermore, the number of treated patients is limited and the incidence of acute and chronic GvHD is lower, resulting in a very heterogeneous approach to this complication by pediatric hematologists. Some conventional therapies and anti-cytokine monoclonal antibodies used in the adult setting have been evaluated in children. In recent years, the increasing understanding of the biological mechanisms underpinning the pathogenesis of GvHD justified the efforts toward the adoption of targeted therapies and non-pharmacologic approaches, with higher response rates and lower immunosuppressive effects. Moreover, many questions regarding the precise timing and setting in which to integrate these new approaches remain unanswered. This Review aims to critically explore the current evidence regarding novel approaches to treat SR-GvHD in pediatric HSCT recipients.

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Budget impact analysis of belumosudil for chronic graft-versus-host disease treatment in the United States

Cited by 3 publications

References 16 publications

Novel therapies for graft versus host disease with a focus on cell therapies

Novel therapies for graft versus host disease with a focus on cell therapies

Navigating the Complexity of Chronic Graft-vs-Host Disease: Canadian Insights into Real-World Treatment Sequencing

Treatment of steroid-refractory graft versus host disease in children

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