Feasibility of a Smartphone Application (App) Pictorial Blood Assessment Chart (PBAC) As an Endpoint in a Von Willebrand Disease Trial

Background: von Willebrand disease (VWD) is a common inherited bleeding disorder characterized by mucosal bleeding. Among affected women, up to 80% have heavy menstrual bleeding, which causes serious medical and psychological morbidity associated with iron deficiency, including reduced physical functioning, anxiety, depression, and poor quality of life. Standard therapies such as hormonal agents and desmopressin are poorly tolerated, and thus the lack of effective therapies for menorrhagia remains a major unmet healthcare need in women with VWD. We designed a prospective, multicenter randomized clinical trial, Von Willebrand Disease Minimize Menorrhage (VWDMin) Trial to compare recombinant VWF (Vonvendi®) with the current recommended non-hormonal agent, tranexamic acid (Lysteda®) . To determine the practicality, usefulness, and satisfaction of a mobile Smartphone Application (App) to gather bleeding data for this clinical trial, we conducted a cross-over study in women with VWD.

Methods: We piloted a Pictorial Blood Assessment Chart (PBAC) App in women with type I von Willebrand disease (VWD) between 18 and 45 years of age. Women were randomized to one menstrual cycle using the Sisterhood App (http://www.sisterhoodapp.com/), a free commercial app developed by the Hemophilia Federation of America (HFA), or to PBAC by paper form, then crossed-over to the alternate PBAC for the next cycle. Participants downloaded the App to their own mobile devices and created an account with a username, email address, and password. Per IRB requirements, subjects did not send data via their personal emails or through the App directly, and instead used a dedicated study email account on an institutional server to maintain security and confidentiality. Blood loss data were tracked during monthly cycles by PBAC, a validated assessment tool for menstrual blood loss. At the completion of the two cycles, subjects completed an online survey (www.surveymonkey.com/r/8K9FGMG) regarding their experience with the App, including simplicity, accuracy, feasibility, and usability, as compared with paper form.

Results: Thirteen women were randomized to paper or App between August and December 2016. Of these, 5 completed two cycles and the post-study survey. Of the remaining women, 1 withdrew from the study due to pregnancy, 1 withdrew due to difficulties with the App, and 6 were lost to follow-up. Among those who completed the post-study survey, 3 preferred the App and 2 preferred the paper form. Generally, participants liked the convenience of the App and additional App features such as symptom trackers and reports that could be sent directly to their physicians. All subjects felt the App was more or equally accurate compared with paper form. However, some noted occasional problems with the App freezing, and several cited the difficulty of setting up and using a separate study email required for security to send App data as a major obstacle to completing the study.

Discussion: Mobile phone apps are convenient, accessible, widely used by patients, and demonstrate potential usefulness as tools for collecting clinical data. Our pilot study shows the Sisterhood App may be more convenient, preferred by patients, and at least equally accurate to paper PBAC for recording blood loss data in women with VWD. However, significant patient attrition, which may be in part due to the difficulty of setting up and using a secure institutional email to transmit App data, limits the utility of our results. IRB standards for data collection protect the confidentiality of patient information, but in our study may have made participation prohibitively difficult. Alternative methods of transmitting app-based data are needed before the Sisterhood App can be feasibly used to collect data for clinical studies.