Phase-1/-2 study of pomalidomide in chronic GvHD

Pusic, Iskra; Rettig, Michael P.; DiPersio, John F.; Bauer, Sonja; McFarland, Kyle; Gale, Robert Peter; Pavletić, Steven Z.

doi:10.1038/bmt.2015.298

Cited by 13 publications

(11 citation statements)

References 13 publications

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“…However, doses expected to be effective were poorly tolerated because of somnolence, neuropathy, and constipation. Pomalidomide is a new immune-modulating drug, with a 4000-fold greater inhibition of TNFα relative to thalidomide, and is well tolerated, without the adverse effects commonly seen with the latter 149 . Several features of pomalidomide suggest it may be useful in treating cGVHD 149 .…”

Section: Other Drugsmentioning

confidence: 99%

“…Pomalidomide is a new immune-modulating drug, with a 4000-fold greater inhibition of TNFα relative to thalidomide, and is well tolerated, without the adverse effects commonly seen with the latter 149 . Several features of pomalidomide suggest it may be useful in treating cGVHD 149 . In a phase II, open label, randomized study, patients with moderate/severe unresponsive or progressive cGVHD exhibited an ORR of 47% at 6 months, with a greater response rate in joint/fascia, followed by skin, GVHD 149 .…”

Section: Other Drugsmentioning

confidence: 99%

“…Several features of pomalidomide suggest it may be useful in treating cGVHD 149 . In a phase II, open label, randomized study, patients with moderate/severe unresponsive or progressive cGVHD exhibited an ORR of 47% at 6 months, with a greater response rate in joint/fascia, followed by skin, GVHD 149 . Further studies may help elucidate its potential role in this setting.…”

Section: Other Drugsmentioning

confidence: 99%

See 2 more Smart Citations

Acute and Chronic Graft-Versus-Host-Disease – A Focus on Pediatric Patients

Macedo¹,

Garcia

Gouveia

et al. 2021

jbmtct

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Graft-versus-host disease (GVHD), either in its acute or chronic form, is the main contributory factor for morbidity and non-relapse mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Recent advancements in the classification of this disease, with better applicability and reproducibility of standardized criteria, coupled with improvements in the management of steroid-refractory or resistant cases, have led to promising results. In 2020, the Brazilian Group for Pediatric Bone Marrow Transplantation of the Brazilian Society for Blood and Marrow Transplantation and Cellular Therapy (SBTMO) convened a task force to provide updated, evidence-based guidance for the diagnosis, classification, staging, prophylaxis, and treatment of GVHD, with a focus on the pediatric population, the results of which are presented here.

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Section: Other Drugsmentioning

confidence: 99%

Section: Other Drugsmentioning

confidence: 99%

Section: Other Drugsmentioning

confidence: 99%

See 1 more Smart Citation

Acute and Chronic Graft-Versus-Host-Disease – A Focus on Pediatric Patients

Macedo¹,

Garcia

Gouveia

et al. 2021

jbmtct

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“…Pomalidomide is a member of the class, which is 100‐fold more potent than thalidomide in inhibiting production of TNFα and increasing Th1‐cells and has less associated toxicity. The efficacy and safety of pomalidomide in patients with corticosteroid‐resistant cGVHD have not been extensively studies, but preliminary data suggest some clinical activity in cutaneomucosal cGVHD . Clinical Trials: and .…”

Section: Cellular Therapiesmentioning

confidence: 99%

Advances in the treatment of chronic graft‐versus‐host disease

Johnson

Couriel

2019

Adv Cell Gene Ther

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Chronic Graft Versus Host Disease (cGVHD) occurs in over 50-70% of patients undergoing hematopoietic stem-cell transplantation (HSCT) and is the leading cause of late non-relapse mortality. cGVHD typically has insidious multi-organ involvement and has been associated with a worse quality of life, functional status, and increased risk of subsequent comorbidities. The last several years have seen advances in the understanding of the disease, which provided a framework for the design of translational and clinical studies with newer agents currently at different phases which that may hopefully change the course of the disease. This review provides an overview of more commonly used and newer second line options for the management of cGVHD.

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“…The initial search of electronic databases based on MeSH terms identified studies for tacrolimus (189), sirolimus (82), rituximab (122), ruxolitinib (3), hydroxychloroquine (65), imatinib (78), bortezomib (6), ibrutinib (1), ECP (63), pomalidomide (3), and methotrexate (98). Following the review of these, based on the selection criteria, we included studies of tacrolimus (2) [26,27], sirolimus (3) [28][29][30], rituximab (7) [31][32][33][34][35][36][37], ruxolitinib (1) [38], hydroxychloroquine (1) [39], imatinib (6) [40][41][42][43][44][45], bortezomib (1) [46], ibrutinib (1) [47], ECP (15) [48][49][50][51][52][53][54][55][56][57][58][59][60][61][62], pomalidomide (1) [63], and methotrexate (3) [64][65][66] (eTable 1 in the Supplement). Among these, only 1 randomized controlled trial was identified [52].…”

Section: Selected Studiesmentioning

confidence: 99%

Steroid Refractory Chronic Graft-Versus-Host Disease: Cost-Effectiveness Analysis

Yalniz

Murad

Lee

et al. 2018

Biology of Blood and Marrow Transplantation

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Given the increasing incidence of chronic graft-versus-host disease (cGVHD) and its rapidly escalating costs due to many lines of drug treatments, we aimed to perform a meta-analysis to assess the comparative effectiveness of various treatment options. Using these results, we then conducted a cost-effectiveness analysis for the frequently utilized agents in steroid-refractory cGVHD. We searched for studies examining tacrolimus, sirolimus, rituximab, ruxolitinib, hydroxychloroquine, imatinib, bortezomib, ibrutinib, extracorporeal photopheresis, pomalidomide, and methotrexate. Studies with a median follow-up period shorter than 6 months and enrolling fewer than 5 patients were excluded. Meta-analysis for overall and organ system-specific GVHD response (overall response [ORR], complete response [CR], and partial response [PR]) was conducted for each intervention. Cost per CR and cost per CR + PR were calculated as the quotient of the 6-month direct treatment cost by CR and CR + PR. Forty-one studies involving 1047 patients were included. CR rates ranged from 7% to 30% with rituximab and methotrexate, respectively, and ORR ranged from 30% to 85% with tacrolimus and ruxolitinib, respectively. Cost per CR ranged from US$1,187,657 with ruxolitinib to US$680 with methotrexate. Cost per ORR ranged from US$453 for methotrexate to US$242,236 for ibrutinib. The most cost-effective strategy was methotrexate for all of the organ systems. Pomalidomide was found to be the least cost-effective treatment for eye, gastrointestinal, fascia/joint, skin, and oral GVHD, and imatinib was found to be the least cost-effective treatment for liver and extracorporeal photopheresis for lung GVHD. We observed huge cost-effectiveness differences among available agents. Attention to economic issues when treating cGVHD is important to recommend how treatments should be sequenced, knowing that many patients will cycle through available agents.

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Phase-1/-2 study of pomalidomide in chronic GvHD

Cited by 13 publications

References 13 publications

Acute and Chronic Graft-Versus-Host-Disease – A Focus on Pediatric Patients

Acute and Chronic Graft-Versus-Host-Disease – A Focus on Pediatric Patients

Advances in the treatment of chronic graft‐versus‐host disease

Steroid Refractory Chronic Graft-Versus-Host Disease: Cost-Effectiveness Analysis

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