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Thromb Haemost 2011; 106(04): 641-645
DOI: 10.1160/TH11-04-0274
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

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

Margaret V. Ragni
1   Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA
2   Hemophilia Center of Western Pennsylvania, Pittsburgh, Pennsylvania, USA
,
Rachel C. Jankowitz
1   Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, USA
,
Kristen Jaworski
2   Hemophilia Center of Western Pennsylvania, Pittsburgh, Pennsylvania, USA
,
Elizabeth P. Merricks
3   Department of Pathology and Laboratory Medicine/ Francis Owen Blood Research Laboratory, University of North Carolina, Chapel Hill, North Carolina, USA
,
Mark T. Kloos
3   Department of Pathology and Laboratory Medicine/ Francis Owen Blood Research Laboratory, University of North Carolina, Chapel Hill, North Carolina, USA
,
Timothy C. Nichols
3   Department of Pathology and Laboratory Medicine/ Francis Owen Blood Research Laboratory, University of North Carolina, Chapel Hill, North Carolina, USA
› Author Affiliations Financial support: This study was supported by HHS Federal Region III Hemophilia Treatment Centers, Grant #1-H-30-MC-00038–01; the Center for Diseases Control Prevention of Complications of Hemophilia Grant U10DD000193; the Pennsylvania Department of Health State SAP #04100000330 (MVR); and CTRC/CTSI grant: NIH/NCRR/CTSA UL-1 RR024153; Wyeth, Inc., Grant #102344; and Clinical Trials.gov NCT 00524342.
Further Information

Publication History

Received: 27 April 2011

Accepted after major revision: 08 July 2011

Publication Date:
15 December 2017 (online)

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Summary

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®) 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.

Keywords

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

Notes

* Clinical trial registration number: NCT 00524342.


 

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