Vedolizumab in highly resistant acute gastrointestinal graft-versus-host disease after allogeneic stem cell transplantation: A single-center pediatric series

Rosa, Monika; Jarmoliński, Tomasz; Miśkiewicz-Migoń, Izabella; Liszka, Karolina; Miśkiewicz‐Bujna, Justyna; Panasiuk, Anna; Frączkiewicz, Jowita; Ussowicz, Marek

doi:10.17219/acem/146321

Cited by 7 publications

(5 citation statements)

References 38 publications

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“…In this study, only one patient had non‐GI manifestations, and all patients rapidly achieved durable clinical response and gradual histologic improvement. A similar response profile was reported in a few cases of pediatric patients published so far 10–13 . However, other case series that included adult patients with more severe disease, and with concurrent skin and liver involvement, showed less encouraging results 19,20 .…”

Section: Discussionsupporting

confidence: 79%

“…Evidence of safety and efficacy of this modality for treating GI GVHD in children is scarce with only one case report and three case series recently published. [10][11][12][13] In this study, we present our experience in treating thirteen pediatric patients with vedolizumab for GI aGVHD. Acute GVHD International Consortium (MAGIC) staging criteria.…”

Section: Stagementioning

confidence: 99%

“…A similar response profile was reported in a few cases of pediatric patients published so far. [10][11][12][13] However, other case series that included adult patients with more severe disease, and with concurrent skin and liver involvement, showed less encouraging results. advantageous in these high-risk patients, where immune suppression may hamper the desirable graft-versus-leukemia effect.…”

Section: Chryseobacterium Gambrini 1 (Blood)mentioning

confidence: 99%

“…Following recent reports of safe and efficacious treatment with vedolizumab for adult patients with aGVHD and reports of successful treatment in children with IBD, we started to treat selected pediatric patients, who presented with predominant GI manifestations of aGVHD, with vedolizumab. Evidence of safety and efficacy of this modality for treating GI GVHD in children is scarce with only one case report and three case series recently published 10–13 . In this study, we present our experience in treating thirteen pediatric patients with vedolizumab for GI aGVHD.…”

Section: Introductionmentioning

confidence: 96%

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Vedolizumab for pediatric patients with gastrointestinal acute graft‐versus‐host‐disease

Bier

Zaidman

Schejter

et al. 2022

Pediatric Blood & Cancer

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Vedolizumab, an anti-α4β7 integrin monoclonal antibody, impairs homing of T-cells to the gastrointestinal (GI) endothelium and acts as a gut-selective anti-inflammatory agent. Recent reports of the efficacy of vedolizumab in treating lower GI acute graftversus-host disease (aGVHD) are promising, but experience in children is scarce. We present a cohort of 13 pediatric patients who were treated with vedolizumab for GI aGVHD. Ten of the patients were treated for steroid-refractory disease, out of which, six suffered from severe (stage 3 or 4) GI disease before the first dose of vedolizumab. In the other three patients, vedolizumab was introduced early in the disease course. Median time between GI GVHD onset to vedolizumab treatment was 23 days (range 7-59 days), with a median of 3 doses (range 1-5) per patient. GI GVHD staging was evaluated at various time points after the first vedolizumab dose, showing improvement in nine of the 13 patients. After a median follow-up time of 13 months (range 6-34 months), eight patients completely recovered, two had ongoing chronic colitis, and three patients died. During the vedolizumab treatment period, 38 infectious episodes were noted, most of them GI related. The unique activity profile of vedolizumab makes it an appealing treatment option for lower GI aGVHD, but caution for concurrent infections is warranted.

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Section: Discussionsupporting

confidence: 79%

Section: Stagementioning

confidence: 99%

Section: Chryseobacterium Gambrini 1 (Blood)mentioning

confidence: 99%

Section: Introductionmentioning

confidence: 96%

See 2 more Smart Citations

Vedolizumab for pediatric patients with gastrointestinal acute graft‐versus‐host‐disease

Bier

Zaidman

Schejter

et al. 2022

Pediatric Blood & Cancer

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“…Two of these patients experienced a complete response ( 115 ). Rosa et al reported 3 pediatric patients with oncological diseases and grade IV gut aGvHD, of whom only one achieved GvHD remission and Fukuta et al reported 1 patient with a clinical response to vedolizumab ( 116 , 117 ). Aldouby Bier reported on 13 pediatric patients with SR gut aGvHD treated with vedolizumab, among whom 8 presented a clinical recovery and 2 had ongoing chronic colitis.…”

Section: Monoclonal Antibodiesmentioning

confidence: 99%

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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Recent advances in acute gastrointestinal graft versus host disease (aGvHD): aspects of steroid-resistant disease

Kujawska,

Zeiser,

Gil

2024

Ann Hematol

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Acute Graft versus Host Disease (aGvHD) is a common immunological complication occurring in patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Moreover, aGvHD is associated with a higher risk of infections and metabolic complications, affecting non-relapse mortality. Progress in transplantation has changed the prophylactic and therapeutic strategies of aGvHD and improved patient outcomes. The standard first-line therapy remains steroids, with a response rate of about 50%. The Janus Kinase 2 (JAK2) inhibitor, ruxolitinib, is an effective second-line therapy. The management of patients who developed a disease that is refractory to steroids and ruxolitinib, especially in the severe gastrointestinal forms of aGvHD, is not validated and remains an unmet medical need. In the article, we present the current clinical practice, as well as the latest advances targeting pathophysiological pathways of GvHD and gut microbiota, which may be a potential future of aGvHD therapy.

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Vedolizumab in highly resistant acute gastrointestinal graft-versus-host disease after allogeneic stem cell transplantation: A single-center pediatric series

Cited by 7 publications

References 38 publications

Vedolizumab for pediatric patients with gastrointestinal acute graft‐versus‐host‐disease

Vedolizumab for pediatric patients with gastrointestinal acute graft‐versus‐host‐disease

Treatment of steroid-refractory graft versus host disease in children

Recent advances in acute gastrointestinal graft versus host disease (aGvHD): aspects of steroid-resistant disease

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