Rituximab for eradicating inhibitors in people with acquired haemophilia A

Review question

We reviewed the evidence about the effect of rituximab for treating people with acquired haemophilia A.

Background

Acquired hemophilia A is a rare but severe bleeding disorder caused by the autoantibody directed against factor VIII (FVIII, a blood clotting protein) in people with no previous history of a bleeding disorder. This bleeding disorder occurs more frequently in the elderly and may be associated with several other conditions (e.g. solid tumours and autoimmune diseases), or with drugs and is sometimes associated with pregnancy. However, in about half of the cases the causes are unknown. Bleeding occurs in the skin, mucous membranes, and muscles; with joint bleeds being unusual. The goals of management are to stop acute bleeding episodes and to eliminate factor VIII autoantibodies. Corticosteroid treatment (prednisone), with or without, cyclophosphamide is regarded by most clinicians as the most effective standard first line intervention for eradicating inhibitors; however, up to one third of people do not respond to this therapy.

Search date

The evidence is current to: 01 March 2016.

Key results

We were not able to identify any randomised controlled trials to include in this review. We have not been able to draw a definitive conclusion on the best available treatment. Randomised controlled trials are needed to evaluate the precise role of rituximab in acquired hemophilia A, but the rarity of the condition is hindering their planning and execution. While waiting for better evidence, people with haemophilia and clinicians need to base treatment decisions on the largest and better conducted observational studies.

Authors' conclusions: 

No randomised clinical trials of rituximab for acquired hemophilia A were found. Thus, based on the highest quality of evidence, we are not able to draw any conclusions or make any recommendations on rituximab for eradicating inhibitors in people with acquired haemophilia A. Given that undertaking randomised controlled trials in this field is a complex task, the authors suggest that, while planning such trials, clinicians treating the disease continue to base their choices on alternative, lower quality sources of evidence. The authors plan, for a future update of this review, to appraise and incorporate any randomised controlled trials, as well as other high-quality non-randomised studies.

Read the full abstract...
Background: 

Acquired haemophilia A is a rare bleeding disorder caused by the development of specific autoantibodies against coagulation factor VIII. Rituximab may be an alternative approach to the treatment of acquired haemophilia by eradicating FVIII autoantibodies.

Objectives: 

To assess and summarise the efficacy and adverse effects of rituximab for treating people with acquired haemophilia A.

Search strategy: 

We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's trials registers, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and conference proceedings.

Date of last search of the Cochrane Cystic Fibrosis and Genetic Disorders Group's trials registers: 01 March 2016.

Selection criteria: 

Randomised and quasi-randomised controlled trials of rituximab for people with acquired hemophilia A, with no restrictions on gender, age or ethnicity.

Data collection and analysis: 

No trials matching the selection criteria were eligible for inclusion.

Main results: 

No trials matching the selection criteria were eligible for inclusion.

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