Original Research
Outpatient Management of Heavy Menstrual Bleeding in Adolescent and Young Women with Inherited Platelet Function Disorders

https://doi.org/10.1016/j.jpag.2020.06.019Get rights and content

Abstract

Study Objective

To assess the treatment patterns and efficacy of hormonal (HM) and non-HM (NHM) management of heavy menstrual bleeding (HMB) in young women with inherited platelet function disorders (IPFDs).

Design, Setting, and Participants

A retrospective chart review was performed of outpatient treatment of HMB in female patients age 9-25 years who were diagnosed with IPFDs and referred to gynecology and/or hematology at a tertiary care hospital between 2006 and 2018.

Interventions

The study sample was identified using billing codes for IPFDs. Data on HM and NHM treatments and outcomes over a one- to two-year period were collected. Initial treatment was defined as the first treatment prescribed after referral. Descriptive statistics, Pearson χ2, and t tests were used for analysis.

Main Outcome Measures

Treatment failure was defined as a change in treatment method because of continued bleeding.

Results

Thirty-four girls met inclusion criteria. After their initial visit, 19/34 (56%) were treated with HM, 12/34 (35%) with NHM, 2/34 (6%) with a combination of methods, and 1/34 (3%) were untreated. Initial treatment failed in 19/34 (56%) and those patients subsequently required a mean of 2 additional treatments during follow-up. Of the 34 included, 6/34 (18%) remained uncontrolled despite numerous treatment changes and 2/34 (6%) because of noncompliance. When control was achieved, 7/26 (27%) of patients were receiving combined oral contraceptives and 6/26 (23%) desmopressin acetate.

Conclusion

HMB in girls with IPFDs can be difficult to control despite ongoing follow-up and treatment changes. Although the most effective treatment for HMB in young women with IPFDs was not identified, these findings will help providers and patients with setting expectations. Prospective studies are needed to develop recommendations on best practices.

Introduction

Heavy menstrual bleeding (HMB) is a common presenting concern of adolescent patients that can affect adolescents significantly, from iron deficiency leading to anemia to inability to participate in activities. HMB is often defined as menstrual blood loss greater than 80 cc or, clinically, as bleeding that: lasts more than seven days, soaks through one or more tampons or pads every hour for several consecutive hours, requires more than one pad at a time or pad/tampon changes overnight, or is associated with clots that are quarter-sized or larger. Etiology of HMB in adolescents includes anovulatory bleeding due to prematurity of the hypothalamic-pituitary-ovarian axis as well as inherited bleeding disorders. In adolescents who present with HMB, prevalence of inherited bleeding disorders, including platelet function disorders, is similar to that in the adult women population (2%-44% in adolescents, compared with <1% to 47% in adult women).1,2 HMB is prevalent among women with bleeding disorders, ranging from 51-98% and varying by diagnosis.2

Inherited platelet function disorders (IPFDs) are a group of individually rare, clinically heterogeneous inherited disorders of platelet function. IPFDs have been reported as a common cause of HMB in adolescents with prevalence rates ranging from 2% to 47%.3,4 A recent study showed IPFDs in nearly half of women who presented with HMB who do not have von Willebrand disease.5

The goals of medical treatment of HMB are: (1) decreasing menstrual flow, or complete menstrual suppression; and (2) correcting iron deficiency and anemia. Common treatments for adolescents with inherited bleeding disorders include hormonal (HM) therapy (combined estrogen-progestin and progestin-only methods) as well as nonhormonal (NHM) therapies (specific factor therapies, antifibrinolytic drugs [ε-aminocaproic acid or tranexamic acid], and desmopressin acetate [DDAVP]).6

In a recent study Alaqzam et al., who investigated treatment of HMB in 73 adolescent patients with (46% total, 36% von Willebrand disease, and 10% platelet function disorder) and without bleeding disorders (54%), reported that the levonorgestrel intrauterine device (IUD) achieved the highest rate of menstrual suppression followed by oral norethindrone acetate and the transdermal patch.1 Good menstrual suppression was achieved in patients who used HM therapy in conjunction with the antifibrinolytic tranexamic acid.1 In this study, only 8 patients had platelet function disorder.1

Anecdotal experiences suggest that HMB in patients with IPFDs is difficult to control and optimal choice of treatment for HMB in adolescents with IPFDs is unknown. The current study was, therefore, designed to assess the treatment patterns and efficacy of HM and NHM management of HMB in young women with IPFDs.

Section snippets

Materials and Methods

A retrospective chart review was conducted of female patients age 9-25 years with IPFDs treated for HMB with at least one year of follow-up in the outpatient setting by pediatric gynecology, hematology, or both, at a tertiary care hospital's clinics between 2006 and 2018. The study sample was identified using International Classification of Diseases 9th and 10th revision billing codes for qualitative IPFDs (qualitative platelet defects, D69.1; qualitative platelet defects 287.1).

Diagnostic

Results

Thirty-four girls with IPFD and HMB were included in the study. All patients had at least one year of follow-up, and 28/34 (82%) of patients had two years of follow-up. Demographic characteristics are listed in Table 1. Although the mean hemoglobin level upon presentation was normal, there were 4/34 (12%) of the young women who had a hemoglobin level below 11 g/dL. Only six patients had a history of red blood cell transfusion, most of whom (n = 5) received a blood transfusion for a gynecologic

Discussion

This study illustrates the difficulty of treating HMB in young women with IPFDs. Although HMB is typically successfully treated with HM methods in the general population of women without bleeding disorders, it is clear that women with IPFDs present a greater challenge. Despite an average of one previous treatment before consultation with pediatric hematology or gynecology, IT failed in 56% of patients with platelet disorders, a rate similar to that seen in studies on patients with a variety of

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A.C.W. has been an investigator for trials sponsored by Pfizer, Takeda, Sanofi, and Novo Nordisk, with previous research funding from Novo Nordisk and Shire, and has previously served on medical advisory boards for Shire, Bayer, Aptevo, and Kedrion. The remaining authors indicate no conflicts of interest.

These data were presented, in part, at the North American Society for Pediatric and Adolescent Gynecology 33rd Annual Clinical & Research Meeting, April 11-13, 2019 in New Orleans, Louisiana.

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© 2020 North American Society for Pediatric and Adolescent Gynecology. Published by Elsevier Inc.