Drug updated on 4/16/2024
| Dosage Form | Injection (intravenous; 300 mg/30 mL (10 mg/mL)) |
| Drug Class | Complement inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- The treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.
- The treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (1.2). Limitation of Use Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
- The treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive.
- The treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
Summary
- Eculizumab (Soliris) is a drug used for the treatment of various conditions including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive, and neuromyelitis optica spectrum disorder in adult patients who are anti-aquaporin-4 antibody positive.
- In treating transplant-associated thrombotic microangiopathy (TA-TMA) - a dangerous complication in hematopoietic stem cell transplantation patients - eculizumab has shown to improve overall response rate and survival rate with infection being the most common adverse event reported.
- For PNH patients, eculizumab significantly decreased lactate dehydrogenase levels and transfusion rates while increasing hemoglobin levels over time; however, it was associated with frequent adverse events such as nasopharyngitis, headache, upper respiratory tract infection among others.
- When used as an adjunctive therapy for lupus nephritis complicated by thrombotic microangiopathy secondary to LN or part of diagnosis of aHUS, 93% of treated SLE patients had favorable outcomes from using eculizumab; though further evidence is required to determine its benefits on refractory LN cases without TMA features.
- A case study showed that catastrophic antiphospholipid syndrome patient refractory to conventional therapies responded positively when treated with eculizumab which halted acute progressive thrombotic events and prevented further clinical episodes over one year period suggesting potential use against this condition despite need for more investigation into this approach.
- The information provided above comes from four systematic reviews/meta-analyses documents focusing on different indications where Soliris can be applied effectively albeit some side effects were noted across all studies reviewed.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Soliris (eculizumab) Prescribing Information. | 2020 | Alexion Pharmaceuticals, Inc., Boston, MA, USA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy and safety of eculizumab in the treatment of transplant-associated thrombotic microangiopathy: a systematic review and meta-analysis. | 2021 | Frontiers in Immunology |
| Efficacy and safety of eculizumab for paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis. | 2021 | Journal of Pediatric Hematology Oncology |
| A systematic review of the role of eculizumab in systemic lupus erythematosus-associated thrombotic microangiopathy. | 2020 | BMC Nephrology |
| Eculizumab in refractory catastrophic antiphospholipid syndrome: a case report and systematic review of the literature. | 2019 | Clinical and Experimental Medicine |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Hemolytic uremic syndrome in a developing country: consensus guidelines. | 2019 | Pediatric Nephrology |