Haemophilia (2014), 20, 249–254
DOI: 10.1111/hae.12293
ORIGINAL ARTICLE Rare bleeding disorders
Platelet function defects in adolescents with heavy menstrual bleeding H. L. MILLS,*†1 M. S. ABDEL-BAKI,*†1 J. TERUYA,*†‡ J. E. DIETRICH,*§ M. D. SHAH,*† D . M A H O N E Y J R . , * † D . L . Y E E * † and L . V . S R I V A T H S * † *Department of Pediatrics, Baylor College of Medicine, Houston, TX; †Section of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX; ‡Division of Transfusion Medicine, Baylor College of Medicine, Houston, TX; and §Department of Obstetrics & Gynecology, Baylor College of Medicine, Houston, TX, USA
Summary. Platelet function defects (PFD) are reported to occur frequently in adult women with heavy menstrual bleeding (HMB). Few studies on adolescent HMB report varying incidence rates (2–44%) for PFD. We reviewed our institutional experience in detecting and managing PFD in adolescent HMB. Postmenarchial girls and adolescents with HMB seen at our institution undergo a comprehensive bleeding disorder work-up by paediatric haematology and paediatric gynaecology providers. Whole blood platelet aggregometry (WBPA) is performed as a second tier test after excluding thrombocytopaenia, coagulation factor deficiencies and Von Willebrand disease (VWD). We retrospectively reviewed the medical records of adolescents with HMB seen between June 2009 and November 2010, as approved by the Institutional Review Board. Patient demographics, clinical features, laboratory results, therapy details and patient outcome information were
analysed. Overall, 114 postmenarchial girls and adolescents with HMB were evaluated; 68 patients (59%) had WBPA study performed. Nineteen patients (28%) had at least one aggregation or secretion defect; 12 (18%) had two or more such defects. Treatment included hormonal therapy (13/19; 68%), antifibrinolytic agents (8/19; 42%) and intra-nasal DDAVP (3/19; 16%). Thirteen patients (81%) had improved outcome (median follow-up – 15.6 months; range of 1–66 months). In this study, PFD were identified in nearly one-third of girls with HMB, with the majority of these having two or more defects as identified by WBPA. Further prospective studies are needed to better define the prevalence and address appropriate management of HMB and other bleeding complications of PFD in adolescents.
Introduction
to be 2–44%, clotting factor deficiency to be 8–9% and thrombocytopaenia to be 13–20% [2]. Compared to the adult population, there have been far fewer studies in adolescents with HMB reporting on the prevalence of underlying bleeding disorders. Published studies that have addressed PFD as a cause for HMB [3–6] have several limitations including lack of uniform patient population and testing of all HMB patients for PFD, and when tested, lack of employment of optimal testing methodology. To accurately characterize the prevalence of PFD in adolescent HMB, there exists a need for uniform evaluation of a large group of this patient population, by employing standard testing methodology. At our institution, the multispecialty ‘Young Women’s Bleeding Disorders Clinic’ between paediatric haematology and paediatric gynaecology centres provides comprehensive care for a large number of postmenarchial girls with HMB with diagnosed
Platelet function defects (PFD) are reported as a common cause of heavy menstrual bleeding (HMB), in adult women. Miller and colleagues showed that among 232 women diagnosed with HMB, 56% had abnormal platelet function study [1]. While there is limited information about the prevalence of bleeding disorders among adolescents with HMB, the available data show the prevalence of VWD to be 5–36%, PFD Correspondence: Lakshmi Venkat Srivaths, MD, Associate Professor, Department of Pediatrics, Section of Hematology/ Oncology, Texas Children’s Hospital/Baylor College of Medicine, 6701, Fannin Street, Houston, TX 77030, USA. Tel.: (832) 822-1514; fax: (832) 825-0285; e-mail: [email protected] 1
Both authors contributed equally to the study
Accepted after revision 7 October 2013 © 2013 John Wiley & Sons Ltd
Keywords: adolescent, heavy menstrual bleeding, platelet function defect
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bleeding disorders. This presented us with the unique opportunity of evaluating this specific patient population for the presence of PFD. On the basis of the observation in adult women, we hypothesized that PFD has similar prevalence in adolescents with HMB as seen in the adult population. This study also intended to summarize by retrospective analysis, our experience in detecting and managing PFD in the adolescent HMB population seen at our institution.
Methods We retrospectively reviewed the medical records of postmenarchial girls with HMB seen at our haematology centre between June 2009 and November 2010. This study was included under the Menorrhagia Data Registry protocol approved by the Institutional Review Board. Postmenarchial girls with HMB were seen initially by a primary care physician in the community and were then referred to the adolescent gynaecology service and/or haematology centre at Texas Children’s Hospital for further evaluation. Heavy menstrual bleeding was defined as menses lasting more than 7 days, using 8–10 pads and/or tampons per day, and passing clots >1 inch in diameter or pictorial blood assessment chart (PBAC) score greater than 100 [7,8]; patients who met this definition were eligible for the study. Patients were assessed in the paediatric gynaecology and/or paediatric haematology clinic to undergo a bleeding disorder evaluation. The evaluation was conducted stepwise as three tiers of testing, to test patients for common bleeding disorders first then for less common and rare bleeding disorders in the subset of patients who have negative results on the initial tier/s of testing. First tier testing included a complete blood count, prothrombin time, activated partial thromboplastin time, VW panel and fibrinogen level. Repeat testing was typically done for low VW levels for diagnosis confirmation. The whole blood platelet aggregometry (WBPA) was performed as a second tier test after ruling out thrombocytopaenia, coagulation factor deficiencies and VWD. WBPA was also performed in patients with low VW ristocetin cofactor activity (VWF: RCo) between 30 and 50 IU dL 1. Patients with VWF: RCo
DOI: 10.1111/hae.12293
ORIGINAL ARTICLE Rare bleeding disorders
Platelet function defects in adolescents with heavy menstrual bleeding H. L. MILLS,*†1 M. S. ABDEL-BAKI,*†1 J. TERUYA,*†‡ J. E. DIETRICH,*§ M. D. SHAH,*† D . M A H O N E Y J R . , * † D . L . Y E E * † and L . V . S R I V A T H S * † *Department of Pediatrics, Baylor College of Medicine, Houston, TX; †Section of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Houston, TX; ‡Division of Transfusion Medicine, Baylor College of Medicine, Houston, TX; and §Department of Obstetrics & Gynecology, Baylor College of Medicine, Houston, TX, USA
Summary. Platelet function defects (PFD) are reported to occur frequently in adult women with heavy menstrual bleeding (HMB). Few studies on adolescent HMB report varying incidence rates (2–44%) for PFD. We reviewed our institutional experience in detecting and managing PFD in adolescent HMB. Postmenarchial girls and adolescents with HMB seen at our institution undergo a comprehensive bleeding disorder work-up by paediatric haematology and paediatric gynaecology providers. Whole blood platelet aggregometry (WBPA) is performed as a second tier test after excluding thrombocytopaenia, coagulation factor deficiencies and Von Willebrand disease (VWD). We retrospectively reviewed the medical records of adolescents with HMB seen between June 2009 and November 2010, as approved by the Institutional Review Board. Patient demographics, clinical features, laboratory results, therapy details and patient outcome information were
analysed. Overall, 114 postmenarchial girls and adolescents with HMB were evaluated; 68 patients (59%) had WBPA study performed. Nineteen patients (28%) had at least one aggregation or secretion defect; 12 (18%) had two or more such defects. Treatment included hormonal therapy (13/19; 68%), antifibrinolytic agents (8/19; 42%) and intra-nasal DDAVP (3/19; 16%). Thirteen patients (81%) had improved outcome (median follow-up – 15.6 months; range of 1–66 months). In this study, PFD were identified in nearly one-third of girls with HMB, with the majority of these having two or more defects as identified by WBPA. Further prospective studies are needed to better define the prevalence and address appropriate management of HMB and other bleeding complications of PFD in adolescents.
Introduction
to be 2–44%, clotting factor deficiency to be 8–9% and thrombocytopaenia to be 13–20% [2]. Compared to the adult population, there have been far fewer studies in adolescents with HMB reporting on the prevalence of underlying bleeding disorders. Published studies that have addressed PFD as a cause for HMB [3–6] have several limitations including lack of uniform patient population and testing of all HMB patients for PFD, and when tested, lack of employment of optimal testing methodology. To accurately characterize the prevalence of PFD in adolescent HMB, there exists a need for uniform evaluation of a large group of this patient population, by employing standard testing methodology. At our institution, the multispecialty ‘Young Women’s Bleeding Disorders Clinic’ between paediatric haematology and paediatric gynaecology centres provides comprehensive care for a large number of postmenarchial girls with HMB with diagnosed
Platelet function defects (PFD) are reported as a common cause of heavy menstrual bleeding (HMB), in adult women. Miller and colleagues showed that among 232 women diagnosed with HMB, 56% had abnormal platelet function study [1]. While there is limited information about the prevalence of bleeding disorders among adolescents with HMB, the available data show the prevalence of VWD to be 5–36%, PFD Correspondence: Lakshmi Venkat Srivaths, MD, Associate Professor, Department of Pediatrics, Section of Hematology/ Oncology, Texas Children’s Hospital/Baylor College of Medicine, 6701, Fannin Street, Houston, TX 77030, USA. Tel.: (832) 822-1514; fax: (832) 825-0285; e-mail: [email protected] 1
Both authors contributed equally to the study
Accepted after revision 7 October 2013 © 2013 John Wiley & Sons Ltd
Keywords: adolescent, heavy menstrual bleeding, platelet function defect
249
250
H. L. MILLS et al.
bleeding disorders. This presented us with the unique opportunity of evaluating this specific patient population for the presence of PFD. On the basis of the observation in adult women, we hypothesized that PFD has similar prevalence in adolescents with HMB as seen in the adult population. This study also intended to summarize by retrospective analysis, our experience in detecting and managing PFD in the adolescent HMB population seen at our institution.
Methods We retrospectively reviewed the medical records of postmenarchial girls with HMB seen at our haematology centre between June 2009 and November 2010. This study was included under the Menorrhagia Data Registry protocol approved by the Institutional Review Board. Postmenarchial girls with HMB were seen initially by a primary care physician in the community and were then referred to the adolescent gynaecology service and/or haematology centre at Texas Children’s Hospital for further evaluation. Heavy menstrual bleeding was defined as menses lasting more than 7 days, using 8–10 pads and/or tampons per day, and passing clots >1 inch in diameter or pictorial blood assessment chart (PBAC) score greater than 100 [7,8]; patients who met this definition were eligible for the study. Patients were assessed in the paediatric gynaecology and/or paediatric haematology clinic to undergo a bleeding disorder evaluation. The evaluation was conducted stepwise as three tiers of testing, to test patients for common bleeding disorders first then for less common and rare bleeding disorders in the subset of patients who have negative results on the initial tier/s of testing. First tier testing included a complete blood count, prothrombin time, activated partial thromboplastin time, VW panel and fibrinogen level. Repeat testing was typically done for low VW levels for diagnosis confirmation. The whole blood platelet aggregometry (WBPA) was performed as a second tier test after ruling out thrombocytopaenia, coagulation factor deficiencies and VWD. WBPA was also performed in patients with low VW ristocetin cofactor activity (VWF: RCo) between 30 and 50 IU dL 1. Patients with VWF: RCo
