Phase III prospective open-label trial of recombinant interleukin-11 in women with mild von Willebrand disease and refractory menorrhagia

作者：Ragni Margaret V^*; Jankowitz Rachel C; Jaworski Kristen; Merricks Elizabeth P; Kloos Mark T; Nichols Timothy C

来源：Thrombosis and Haemostasis, 2011, 106(4): 641-645.

DOI：10.1160/TH11-04-0274

• Clinical trial
• interleukin-11
• menorrhagia
• von Willebrand disease
• women

Lack of effective treatment for menorrhagia is the greatest unmet healthcare need in women with von Willebrand disease (VWD). We conducted a single-centre phase II clinical trial to determine efficacy and safety of recombinant IL-11 (rhIL-11, Neumega (R)) given subcutaneously for up to seven days during six consecutive menstrual cycles each in seven women with mild VWD and menorrhagia refractory to haemostatic or hormonal agents. rhIL-11 reduced menstrual bleeding severity as measured by pictorial blood assessment chart (PBAC) >= 50% (to <100) in 71% of subjects, cycle severity >= 50% in 71%, and bleeding duration >= 2 days in 85%, all p <= 0.01. After rhIL-11, plasma VWF:RCo increased 1.1-fold, but did not correlate with PBAC, r=0.116, bleeding duration, r=0.318, or cycle severity, r=-0.295, or hsCRP, r=-0.003, all p>0.05. Platelet VWF mRNA expression by quantitative PCR increased mean four-fold (1.0-13.5). rhIL-11 was well tolerated with grade 1 or less fluid retention, flushing, conjunctival erythema, and local bruising. In summary, rhIL-11 reduces menorrhagia safely and warrants further study.

• 出版日期2011-10