A low‐dose rituximab regimen for first‐line treatment of acquired haemophilia A

Abstract

Novelty statement:1. What is the new aspect of your work?• Acquired haemophilia A (AHA) typically occurs in elderly patients with multiple comorbidities. Immunosuppression has demonstrated improved overall survival and remission rates; however, treatment-related complications in this cohort often occur. Efficacious but dose-reduced immunosuppression for this rare condition will hopefully reduce morbidity and is an important area for further research. What is the central finding of your work?• Universal immunosuppression in AHA patients improves overall survival, and 87.5% of patients achieved a complete remission in a median of 48 days. The use of upfront rituximab with corticosteroids is increasing and is associated with shorter times to complete remission. Low-dose rituximab (100 mg/m 2 weekly IV for 4 doses) is emerging as an alternative and appears non-inferior in this case series.

Single‐dose rituximab plus glucocorticoid versus cyclophosphamide plus glucocorticoid in patients with newly diagnosed acquired hemophilia A: A multicenter, open‐label, randomized noninferiority trial

“…Various definitions of remission in previous AHA studies and the crossover design in our study may contribute to these results 9 . There are case reports on reduced‐dose RTX regimen showing good efficacy 11–15 . They also showed no serious AEs, especially no severe infection.…”

“…9 There are case reports on reduced-dose RTX regimen showing good efficacy. [11][12][13][14][15] They also showed no serious AEs, especially no severe infection. Complete B-cell depletion was also observed in our study nearly 1 or 2 weeks after RTX administration, demonstrating the efficacy of our single-dose RTX in B-cell depletion.…”

“…The multicenter retrospective China Acquired Hemophilia Registry (CARE) study reported a comparable CR rate between the RTX‐based therapy (91%) and the CTX regimen (88%), and the time to CR was 47 days for the RTX regimen versus 62 days for the CTX regimen 5 . In addition, the reduced‐dose RTX regimen (100 mg/week for 4 weeks or 375 mg/m 2 for one dose) also had good efficacy and low‐grade infection with no report of obvious long‐term adverse events (AEs) in AHA patients 11–16 . Thus, single‐dose RTX therapy would be more suitable as the first choice, especially for young patients in avoid of malignancy and gonadal failure.…”

“…5 In addition, the reduced-dose RTX regimen (100 mg/ week for 4 weeks or 375 mg/m 2 for one dose) also had good efficacy and low-grade infection with no report of obvious long-term adverse events (AEs) in AHA patients. [11][12][13][14][15][16] Thus, single-dose RTX therapy would be more suitable as the first choice, especially for young patients in avoid of malignancy and gonadal failure. However, all the existing conclusions are based on retrospective or small-scale prospective studies and clinical experience.…”

Cyclophosphamide vs Rituximab for Eradicating Inhibitors in Acquired Hemophilia A: A Randomized Trial in 108 Patients

“…Our results are comparable to those of the recent randomized open non inferiority trial which showed similar efficacy and safety of a single dose of RTX [24 (77.4 %) patients in the rituximab group and 22 (68.8 %) in the cyclophosphamide group achieved CR within 8 weeks. However, patients were younger (42 [31,59] for the single-dose rituximab group and 55 [34,67] for the cyclophosphamide group), and an autoimmune disease was the most common underlying disease in both groups [23].…”