Selective studies evaluating TA-TMA therapies
| Published studies . | ||||||
|---|---|---|---|---|---|---|
| Drug . | Author . | Study type . | Number of patients . | Inclusion criteria . | Response . | Survival . |
| Eculizumab | Jodele 2020 | Retrospective, single institution | 64 | Children and young adults with hrTMA (concurrent proteinuria and elevated C5b9) and treated with eculizumab | 64% response 56% CR | OS 77% at 6 months |
| Zhang 2021 | Systemic review with retrospective studies | 116 (including 64 from above) | Studies that included patients diagnosed with TA-TMA and eculizumab used first- or second-line | 71% response 32% CR | OS 52% at last follow-up of each study | |
| Svec 2023 | Retrospective, multi-institution | 82 | Allogeneic transplant patients <18 years with TA-TMA diagnosed by the treatment center and treated with eculizumab | 48% response | OS 47% at 6 months | |
| Jodele 2024 | Prospective, single-arm, multi-institution (NCT03518203) | 21 | Children and young adults, at least 5 kg, meeting criteria for hrTMA (concurrent proteinuria and elevated C5b9) | 67% response 48% CR | OS 71% at 6 months | |
| Narsoplimab | Khaled 2022 | Prospective, single-arm, multi-institution (NCT02222545) | 28 | Adults 18 or older with persistent TA-TMA | 61% response | OS 68% at 100 days |
| Defibrotide | Yeates 2017 | Retrospective, multi-institution | 39 | Pediatric and adult patients who received defibrotide to treat TA-TMA | 77% response | OS 59%-64% at unspecified follow-up |
| Marinez-Munoz 2019 | Retrospective, single institution | 16 | Adult patients who underwent allogeneic HCT with a diagnosis of TA-TMA | 65% response | OS 59% at unspecified follow-up | |
| Published studies . | ||||||
|---|---|---|---|---|---|---|
| Drug . | Author . | Study type . | Number of patients . | Inclusion criteria . | Response . | Survival . |
| Eculizumab | Jodele 2020 | Retrospective, single institution | 64 | Children and young adults with hrTMA (concurrent proteinuria and elevated C5b9) and treated with eculizumab | 64% response 56% CR | OS 77% at 6 months |
| Zhang 2021 | Systemic review with retrospective studies | 116 (including 64 from above) | Studies that included patients diagnosed with TA-TMA and eculizumab used first- or second-line | 71% response 32% CR | OS 52% at last follow-up of each study | |
| Svec 2023 | Retrospective, multi-institution | 82 | Allogeneic transplant patients <18 years with TA-TMA diagnosed by the treatment center and treated with eculizumab | 48% response | OS 47% at 6 months | |
| Jodele 2024 | Prospective, single-arm, multi-institution (NCT03518203) | 21 | Children and young adults, at least 5 kg, meeting criteria for hrTMA (concurrent proteinuria and elevated C5b9) | 67% response 48% CR | OS 71% at 6 months | |
| Narsoplimab | Khaled 2022 | Prospective, single-arm, multi-institution (NCT02222545) | 28 | Adults 18 or older with persistent TA-TMA | 61% response | OS 68% at 100 days |
| Defibrotide | Yeates 2017 | Retrospective, multi-institution | 39 | Pediatric and adult patients who received defibrotide to treat TA-TMA | 77% response | OS 59%-64% at unspecified follow-up |
| Marinez-Munoz 2019 | Retrospective, single institution | 16 | Adult patients who underwent allogeneic HCT with a diagnosis of TA-TMA | 65% response | OS 59% at unspecified follow-up | |
| Ongoing clinical trials . | ||||||
|---|---|---|---|---|---|---|
| Drug . | Sponsor . | Study type . | Number of patients . | Inclusion criteria . | End points to collect . | |
| Ravulizumab | Alexion | Randomized, double-blind, placebo-controlled, multi-institution (NCT04543591) | 106 estimated | 12 years of age or older, HCT in the last 12 months, TMA diagnosis for at least 72 hours, weighing 30 kg or greater | - TMA response at 26 weeks | - OS at 26 and 52 weeks - nonrelapse mortality at 26 and 52 weeks |
| Nomacopan | AKARI Therapeutics | Prospective, single-arm, multi-institution (NCT04784455) | 50 estimated | 6 months to 18 years of age, undergone HCT with a diagnosis of TA-TMA | - Transfusion independence by week 24 | - rUPCR 2 mg/mg or less at 24 weeks |
| Narsoplimab | Omeros | Prospective, single-arm, multi-institution (NCT05855083) | 18 estimated | Pediatric patients 28 days to <18 years of age with high-risk TMA | - % clinical response | - OS at 100 days after high-risk TMA diagnosis |
| Pegcetacoplan | Sobi | Prospective, single-arm, multi-institution (NCT05148299) | 12 estimated | Adults 18 years or older, with a diagnosis of TA-TMA, and with rUPCR 1 mg/mg or greater | - % clinical & TMA responses at 12 and 24 weeks | - OS at 100 days - OS 24 days from treatment start |
| Ongoing clinical trials . | ||||||
|---|---|---|---|---|---|---|
| Drug . | Sponsor . | Study type . | Number of patients . | Inclusion criteria . | End points to collect . | |
| Ravulizumab | Alexion | Randomized, double-blind, placebo-controlled, multi-institution (NCT04543591) | 106 estimated | 12 years of age or older, HCT in the last 12 months, TMA diagnosis for at least 72 hours, weighing 30 kg or greater | - TMA response at 26 weeks | - OS at 26 and 52 weeks - nonrelapse mortality at 26 and 52 weeks |
| Nomacopan | AKARI Therapeutics | Prospective, single-arm, multi-institution (NCT04784455) | 50 estimated | 6 months to 18 years of age, undergone HCT with a diagnosis of TA-TMA | - Transfusion independence by week 24 | - rUPCR 2 mg/mg or less at 24 weeks |
| Narsoplimab | Omeros | Prospective, single-arm, multi-institution (NCT05855083) | 18 estimated | Pediatric patients 28 days to <18 years of age with high-risk TMA | - % clinical response | - OS at 100 days after high-risk TMA diagnosis |
| Pegcetacoplan | Sobi | Prospective, single-arm, multi-institution (NCT05148299) | 12 estimated | Adults 18 years or older, with a diagnosis of TA-TMA, and with rUPCR 1 mg/mg or greater | - % clinical & TMA responses at 12 and 24 weeks | - OS at 100 days - OS 24 days from treatment start |
CR, complete response; HCT, hematopoietic cell transplantation; hrTMA, high-risk TMA; OS, overall survival; rUPCR, random urine protein to creatinine ratio.