Patient Preference and Mobile Application Use in Hemophilia A: A Cross-Sectional Survey from Eastern Europe

Abstract

Hemophilia A, a bleeding disorder caused by a deficiency of clotting factor VIII (FVIII), is commonly treated with FVIII prophylaxis. Mobile applications help personalize prophylactic regimens by creating individual pharmacokinetic (PK) profiles. This study evaluated how patients with hemophilia A use mobile applications for PK-guided prophylaxis and explored preferences and patterns of usage across different age groups. Between May and September 2022, patients from Bulgaria, Czechia, Hungary, Lithuania, and Romania participated in a cross-sectional survey. The survey collected information on demographics, documentation preferences (mobile applications or paper diaries), usage patterns, and user satisfaction. Of 84 participants, 40.5% used either a mobile application or paper diary exclusively. Czechia had the highest application usage (94.1%), followed by Romania (50.0%). While 84.2% of the application users were satisfied, many suggested improvements including better medication tracking and smartwatch compatibility. No correlation was found between age and documentation type or application preference. Mobile applications were perceived useful, but physician engagement was higher with paper diaries. These findings suggest potential to enhance mobile applications for better patient–physician interaction and user experience.

Keywords

factor VIII hemophilia A mobile applications PK-guided prophylaxis

Introduction

Hemophilia A is a rare inherited genetic bleeding disorder caused by insufficient or missing clotting factor VIII (FVIII).^1,2 Worldwide, hemophilia A affects approximately 174 000 people and occurs in approximately one in 5000 male births.³ At-home prophylactic self-infusion of the FVIII concentrate is currently the primary standard of care for preventing bleeding and preserving normal musculoskeletal function to improve the quality of life among patients with hemophilia A.^4,5 However, the pharmacokinetic (PK) profiles of FVIII have high interindividual variability.^6‐8

Personalized prophylaxis may improve bleeding prevention through individualized dose modifications and frequency of FVIII administration, adjusted according to patient bleeding history, individual PK characteristics, physical activity, and lifestyle. Indeed, the World Federation of Haemophilia guidelines recommend targeting FVIII levels (through levels of >3%–5% or higher) based on individual PK profiles, bleeding patterns, and lifestyle factors.⁹ Mobile and web-based applications may therefore represent a promising approach to personalize contemporary FVIII prophylaxis by facilitating simulated dosing regimens using individualized patient PK profiles, which may be established using two to three blood samples collected at 4, 24, and 48 h after FVIII infusion.¹⁰

Several patient-focused mobile applications have been designed to respond to individual patient data, of which the most common are myPKFiT^® (Takeda), myWAPPS (McMaster University), and florio^® HAEMO (Sobi™).^11‐15 Both the florio^® HAEMO and myWAPPS use the WAPPS-Hemo infrastructure, which is a web-accessible, centralized, dedicated service that consists of an actively moderated database of patient PK data.¹⁶ These applications empower patients to live healthier lifestyles, reinforce care provided by healthcare professionals, and promote adherence to individualized management plans.^17‐19 Moreover, mobile applications allow for patient self-care by enabling patients to actively engage in their treatment, interact with and gain actionable knowledge about their care and participate in decision-making processes.^11,12 Therefore, this study aimed to assess the usage of mobile applications commonly utilized in PK-guided interactive hemophilia A prophylaxis and to investigate patients’ preferences and experiences with these applications. Data were also stratified based on geography and age to explore potential regional and demographic differences in the use and preferences for mobile applications and paper diaries.

Materials and Methods

Study Design and Population

This descriptive, observational, survey-based, cross-sectional study, conducted from May to September 2022, assessed patients who were using PK-guided FVIII prophylaxis for hemophilia A management. Patients were recruited through local patient organizations in Bulgaria, Czechia, Hungary, Lithuania, and Romania. Eligible patients were aged ≥12 years, had received a diagnosis of hemophilia A, had been prescribed FVIII for PK-guided prophylaxis, and were willing and able to provide informed consent. Parental or guardian assent was requested for individuals aged <18 years.

A single survey was provided directly to patients through local patient organizations. The survey, which is detailed in Supplemental S1, focused on patient preferences for mobile healthcare applications or written paper diaries for monitoring hemophilia A prophylaxis and collected real-world data on patient demographics, patterns of mobile application use, application satisfaction, application usefulness, and intention for future application use (Supplement S1, Survey questionnaire). Surveys were administered until a total of 80 patient responses were collected from all participating countries. The target of 80 patients was chosen based on practical considerations rather than formal statistical calculations. Given the rarity of hemophilia A and the specific patient population receiving PK-guided prophylaxis, we aimed to include all consecutive patients who met these criteria and provided consent to participate. This approach was informed by feedback from relevant patient associations and the availability of patients within the timeframe of the study. Therefore, the sample size of approximately 80 patients was deemed appropriate for the scope and objectives of the study. A detailed description of the hemophilia-related data collection and its rationale is available in the Table S1 of the supplemental material.

Study Objectives

The primary objective of this study was to gain insight into the utilization of mobile applications by patients receiving prophylactic treatment for hemophilia A. The secondary objective was to describe patient populations using mobile applications and evaluate the reasons for discontinuation or declining use of mobile applications. The exploratory objective was to assess the potential correlations between the use of mobile device applications and patient characteristics.

Study Outcomes

The primary outcome of this study was to investigate patient-reported patterns of application utilization, including the reasons for choosing to use, not to use or to discontinue the use of mobile applications, as well as changing the application selected. Overall satisfaction with mobile applications was assessed through patients’ perception of their ability to manage and calculate FVIII levels, manage and log infusions, track bleeds and symptoms, manage physical activity levels, transfer and review treatment and disease control with healthcare providers, and connect the application with smartwatches. Secondary outcomes included collecting patients’ demographic and clinical characteristic data, determining the correlation between age group and the form of document management used, determining the correlation between age group and mobile application type selected, duration of application use before discontinuation, and advantages of application use. Patients reported disadvantages/concerns on the mobile device applications in the form of feedback and suggestions.

Study Survey

The survey was administered through an electronic data capture (EDC) system (Penta) that was specifically developed based on the questionnaire part of the study protocol and allowed for anonymized data entry. EDC captured data using a mandatory data format and prevented the collection of incomplete data. The surveys were available in local languages of the respective countries. Patients completed the survey in the absence of a healthcare provider. The survey captured the following information:

Patient characteristics (age, sex, duration of hemophilia prophylaxis, and FVIII product used)

Method of prophylaxis documentation (written paper diary, mobile application, Excel spreadsheet, or other [free-form text])

Mobile applications used (florio^® HAEMO, myPKFiT^®, myWAPPS, or other [free text])

Patterns of mobile application use (prior application usage, reason for discontinuation)

Frequency of infusion and bleeds/joint swelling records

Physician engagement with the application

Frequency of checking calculated FVIII levels and management of physical activity)

Advantages of mobile applications

Overall patient satisfaction with mobile applications

Patient prioritization of different aspects of mobile applications

Patient feedback and suggestions for mobile application improvement (free text)

Statistical Analysis

This descriptive analysis did not test a hypothesis and thus did not include a formal sample size calculation. Categorical variables were described as frequencies and percentages (%). Correlations between age group and document management status, mobile application type, or patient suggestions were calculated using Pearson's correlation analysis. Overall patient satisfaction was assessed using a scale ranging from 1 (not satisfied) to 10 (highly satisfied). The distribution of mobile application functionality scores was assessed according to the patient's personal prioritization of mobile application functionality on a scale of 1 (lowest priority) to 10 (highest priority). Statistical analyses were performed using Statistical Package for Social Sciences (SPSS) software, version 24.0. Statistical significance was set at P < .05.

Results

Baseline Demographic Characteristics

A total of 84 eligible patients with hemophilia A who were receiving PK-guided FVIII prophylaxis completed the survey. Most patients were enrolled in Hungary (30/84 [35.7%]), Bulgaria (20/84 [23.8%]), and Czechia (17/84 [20.2%]). Most patients were aged 18–29 years (27/84 [32.1%]), and almost all were male (83/84 [98.8%]); one female patient was enrolled. Most patients (83/84 [98.8%]) commenced prophylactic treatment for hemophilia ≥6 months before completing the survey. The most common FVIII therapeutics were efmoroctocog alfa (16/84 [19.1%]) and rurioctocog alfa pegol (14/84 [16.7%]). Document management was most frequently conducted through a written paper diary only (34/84 [40.5%]) or a mobile application only (34/84 [40.5%], Table 1). Among the patients using a mobile application, florio^® HAEMO was the predominant mobile application used (29/40 [72.5%]), whereas myWAPPS was the least common (3/40 [7.5%]). A combination of documentation methods was used by 7/84 (8.3%) of the participants. Other reported applications included MicroHealth (3/40 [7.5%]). Only 4/84 (4.8%) of patients did not use any form of documentation. The details of the demographic characteristics, medication used, and documentation status of the patients in this study are provided in Table 1.

Table 1.

Demographic Characteristics, Documentation Management Status, Utilization Patterns, and Patient-Reported Advantages.

Variable, n (%)	Patients (N = 84)
Age group, years
12–17	15 (17.9)
18–29	27 (32.1)
30–44	20 (23.8)
45–59	14 (16.7)
60–75	8 (9.5)
Sex
Male	83 (98.8)
Female	1 (1.2)
Geographical location
Bulgaria	20 (23.8)
Czechia	17 (20.2)
Hungary	30 (35.7)
Lithuania	9 (10.7)
Romania	8 (9.5)
First commenced hemophilia prophylaxis
<6 months ago	1 (1.2)
6–12 months ago	2 (2.4)
1–3 years ago	1 (1.2)
3–5 years ago	5 (6.0)
>5 years ago	75 (89.3)
Medicinal products received for hemophilia prophylaxis (manufacturer)
Octocog alfa (Takeda)	4 (4.8)
Rurioctocog alfa pegol (Takeda)	14 (16.7)
Lonoctocog alfa (CSL Behring)	1 (1.2)
Eftrenonacog alfa (Bioverativ Therapeutics)	1 (1.2)
Efmoroctocog alfa (Biogen)	16 (19.1)
Turoctocog alfa pegol (Novo Nordisk)	2 (2.4)
Coagulation FVIII (Grifols)	1 (1.2)
FVIII inhibitor (Takeda)	1 (1.2)
Emicizumab (Genentech)	1 (1.2)
Emicizumab/eptacog alfa (Genentech/Novo Nordisk)	1 (1.2)
von Willebrand factor (Takeda)	5 (6.0)
Damoctocog alfa pegol (Bayer)	9 (10.7)
Octocog alfa (Bayer)	7 (8.3)
Turoctocog alfa (Novo Nordisk)	1 (1.2)
Eptacog alfa (Novo Nordisk)	1 (1.2)
Simoctocog alfa (Octapharma)	9 (10.7)
Coagulation FVIII (Octapharma)	6 (7.2)
Moroctocog alfa (Pfizer)	3 (3.6)
None/unknown	1 (1.2)
Documentation management status
Written paper diary	34 (40.5)
Mobile application	34 (40.5)
Multiple methods^a	7 (8.3)
Excel spreadsheet	4 (4.8)
None	4 (4.8)
Other	1 (1.2)
FVIII infusions recorded in a mobile application/written paper diary within the 2 months before the survey
All infusions recorded	61 (72.6)
Majority of infusions recorded	13 (15.5)
Minority of infusions recorded	4 (4.8)
Not instructed to record (or how to record) infusions	3 (3.6)
Did not/forgot to record infusions	3 (3.6)
Bleeds recorded in a mobile application/written paper diary within the 2 months before the survey
All bleeds recorded	47 (56.0)
Majority of bleeds recorded	5 (6.0)
Not instructed to (or how to) record bleeds	4 (4.8)
Did not/forgot to record bleeds	5 (6.0)
No bleeds experienced	23 (27.4)
Swelling/joint pain recorded in a mobile application/written paper diary within the 2 months before the survey
All swelling/joint pain recorded	30 (35.7)
Majority of swelling/joint pain recorded	7 (8.3)
Minority of swelling/joint pain recorded	2 (2.4)
Not instructed to (or how to) record swelling/joint pain	9 (10.7)
Did not/forgot to record swelling/joint pain	7 (8.3)
No swelling/joint pain experienced	29 (34.5)
Physical exercise/strenuous physical activity recorded in a mobile application/written paper diary within the 2 months before the survey
Regularly recorded exercise or strenuous physical activity	12 (14.3)
Not instructed to (or how to) record physical activity	30 (35.7)
Did not record exercise or strenuous physical activity	32 (38.1)
Mobile application did not allow for recording physical activity	8 (9.5)
No physical exercise or strenuous physical activity due to disease or danger/fear of bleeding	2 (2.4)
Physician engagement with mobile application/written paper diary
Physician reviewed the written paper diary	42 (50.0)
Physician reviewed and/or printed data from the mobile application	19 (22.6)
Patient printed data from the mobile application before the hospital visit	2 (2.4)
Unknown	21 (25.0)
Mobile application previously used to check calculated FVIII levels
florio^® HAEMO	2 (5.0)
WAPPS-Hemo	1 (2.5)
Did not use a mobile application previously	39 (97.5)
Unknown	2 (5.0)
Reasons for previous mobile application discontinuation or switching
Preference for other documentation methods	2 (5.0)
Technical issues	1 (2.5)
Duration of application use before discontinuation
4–6 months	1 (2.5)
>1 year	2 (5.0)
Frequency of checking FVIII levels in the mobile application
More than once daily	2 (5.0)
Less than once daily	31 (77.5)
Only when approaching low FVIII levels	5 (12.5)
Never/almost never	2 (5.0)
Use of FVIII levels in the mobile application to guide physical activity
Calculated FVIII levels used to time physical activity	23 (57.5)
Physical activity performed regardless of FVIII levels	17 (42.5)
Advantages of mobile applications^a
Monitoring FVIII levels and documenting infusions	25
Choosing daily activities according to calculated FVIII levels	25
Physician access to real-time view of symptoms and treatment	22
Provide confidence that FVIII levels are adequate	13
Available in patient's local language	12
Allows patient to feel in control	10
Exporting data from application to share with the healthcare provider	9
Planning sports activities according to calculated FVIII levels	9
Printing the application diary to share with the healthcare provider	4
Other
Calendars and reminders	2
Unspecified	7

^aMultiple responses.

Abbreviation: FVIII: factor VIII.

Written paper diaries were the most common method of document management in Lithuania (6/9 [66.7%]), Bulgaria (13/20 [65.0%]), and Hungary (14/30 [46.7%]), whereas mobile applications were most common in Czechia (16/17 [94.1%]) and Romania (4/8 [50.0%], Table 2). florio^® HAEMO was the most common application used in Bulgaria (4/7 [57.1%]), Czechia (16/17 [94.1%]), and Hungary (7/11 [63.6%]), whereas MicroHealth was most common in Lithuania (1/1 [100.0%]) and myPKFiT^® in Romania (3/4 [75.0%], Table 2).

Table 2.

Document Management Status by Geographical Location and Mobile Application Features.

Patients, n (%)	Bulgaria	Czechia	Hungary	Lithuania	Romania
Total responders	20 (100.0)	17 (100.0)	30 (100.0)	9 (100.0)	8 (100.0)
Written paper diary	13 (65.0)	0 (0)	14 (46.7)	6 (66.7)	1 (12.5)
Mobile application	7 (35.0)	16 (94.1)	6 (20.0)	1 (11.1)	4 (50.0)
Excel spreadsheet	0 (0)	0 (0)	2 (6.7)	0 (0)	2 (25.0)
Multiple methods^a	0 (0)	1 (5.9)	0 (0)	0 (0)	0 (0)
Multiple methods^b	0 (0)	0 (0)	6 (20.0)	1 (11.1)	0 (0)
None	0 (0)	0 (0)	2 (6.7)	0 (0)	1 (12.5)
Other	0 (0)	0 (0)	0 (0)	1 (11.1)	0 (0)
Total mobile application users	7 (100.0)	17 (100.0)	11 (100.0)	1 (100.0)	4 (100.0)
florio^® HAEMO^c	4 (57.1)	16 (94.1)	7 (63.6)	0 (0)	1 (25.0)
myPKFiT^®^d	0 (0)	0 (0)	1 (9.1)	0 (0)	3 (75.0)
WAPPS-Hemo^e	0 (0)	0 (0)	3 (27.3)	0 (0)	0 (0)
Application-electronic log in a clinical trial of a product	1 (14.3)	0 (0)	0 (0)	0 (0)	0 (0)
Other; Microhealth^f	2 (28.6)	0 (0)	0 (0)	1 (100.0)	0 (0)
Multiple methods^a	0 (0)	1 (5.9)	0 (0)	0 (0)	0 (0)

Written paper diary and florio^® HAEMO.

Written paper diary, florio^® HAEMO, and Excel Spreadsheet (N = 3), written paper diary and Excel spreadsheet (N = 1), written paper diary, Excel spreadsheet, and mobile application (N = 1), written paper diary and WAPPS application and Excel spreadsheet (N = 1).

Nonproduct specific features: real-time data exchange with an HCP, record patient-reported data, track physical activity, calculate factor levels, record pain in absence of bleeds, and obtain data from wearable devices.

Product specificity for octocog alfa, rurioctocog alfa pegol: log infusions, view estimated factor levels (both current and future), provide notifications when factor levels are low or infusion is due, record bleeds, check the number of bleed-free days, and export data to share with HCPs.

Nonproduct specific features: assess FVIII levels, calculate future factor levels, track factor levels, and provide notifications when factor levels are low.

Nonproduct specific features: log infusions, track factor stock and refills, track bleeds, track physical activity, track HCP appointments, set reminders, and share with the healthcare team directly.

Abbreviations: FVIII, factor VIII; HCP, healthcare professional.

Utilization of Mobile Applications and Written Paper Diaries

Within 2 months before completing the survey, 61/84 (72.6%) patients reported the recording of all FVIII infusions and 13/84 (15.5%) the recording of the majority of FVIII infusions with a mobile application or written diary (Table 1). Within this timeframe, 47/84 (56.0%) of the patients experienced and recorded all bleeds, and 30/84 (35.7%) experienced and recorded all swelling/joint pain using a mobile application or a written paper diary. Overall, 23/84 (27.4%) of patients did not experience bleeding, and 29/84 (34.5%) of patients did not experience swelling/joint pain during this time frame. Exercise or strenuous physical activity was regularly recorded by 12/84 (14.3%) of the patients using a mobile application or a written paper diary. Instructions to record infusions, bleeds, swelling/joint pain, and physical activity with a mobile application or written paper diary were provided to 3/84 (3.6%), 4/84 (4.8%), 9/84 (10.7%), and 30/84 (35.7%) of the patients, respectively. Overall, physician engagement with written paper diaries was higher than that with mobile applications (42/84 [50.0%] and 19/84 [22.6%], respectively; Table 1). Across countries, physician engagement was highest with written paper diaries in Hungary 25/84 (29.8%), Bulgaria 6/84 (7.1%), Lithuania 5/84 (6.0%), and Romania 3/84 (3.6%) and was highest with mobile applications in Czechia 13/84 (15.5%; Supplemental Table S2). Prior use of mobile applications was reported by 3/44 (6.8%) of patients who discontinued because of technical difficulties or switching to a preferred documentation method. Among patients who were currently using a mobile application, 31/40 (77.5%) used it to check FVIII levels less than once daily, and 23/40 (57.5%) used FVIII levels calculated by the application to guide the timing of physical activity (Table 1).

Patient Satisfaction and Prioritization of Mobile Application Functionality

Of the 40 patients who indicated the specific mobile application used, 27 (67.5%) rated their experience with mobile applications as highly satisfied, with a score of >8 out of a maximum score of 10. The mobile application functionalities most often considered to have the highest priority (a score of 10) were timely logging of infusions (31/40 [77.5%]), calculated future FVIII levels (25/40 [62.5%]), upcoming infusion reminders (24/40 [60.0%]), visible calculated FVIII levels (24/40 [60.0%]), and real-time physician view of treatment and level of disease control (16/40 [40.0%]). The functionality most often considered to have the lowest priority (score of 1) was connection to a smartwatch (14/40 [35.0%]; Figure 1).

Figure 1.

Prioritization of mobile application functionality. It was determined based on a 1–10 scale (1: lowest priority, 10: highest priority). FVIII, factor VIII; HCP, healthcare professional.

Distribution of Document Management Status and Mobile Application Type by Age Group

A written paper diary was most often used by patients aged 18–29 years (16/34 [47.1%]), whereas mobile applications were used most often by patients aged 30–44 years (10/34 [29.4%]), 18–29 years (8/34 [23.5%]), and 12–17 years (7/34 [20.6%]; Table 3). There was no significant correlation between age group and document management status (P = .129). The most common mobile application used across all age groups was florio^® HAEMO. There was no significant correlation between age group and mobile application type (P = .376; Supplemental Table S3).

Table 3.

Distribution of Age Groups According to Document Management Status and Type of Mobile Application.

	Document management status							Type of mobile application
Age (years), n (%)	Written paper diary(N = 34)	Mobile application(N = 34)	Multiple methods(N = 7)	None(N = 4)	Excel Spreadsheet(N = 4)	Other(N = 1)	P-value	florio^® HAEMO(N = 29)	myPKFiT^®(N = 4)	WAPPS-Hemo(N = 3)	P-value
12–17	7 (20.6)	7 (20.6)	–	–	1 (25.0)	–	.129	5 (17.2)	1 (25.0)	–	.376
18–29	16 (47.1)	8 (23.5)	1 (14.3)	1 (25.0)	1 (25.0)	–		7 (24.1)	1 (25.0)	–
30–44	4 (11.8)	10 (29.4)	3 (42.9)	3 (75.0)	–	–		10 (34.5)	1 (25.0)	–
45–59	3 (8.8)	6 (17.6)	2 (28.6)	–	2 (50.0)	1 (100.0)		5 (17.2)	–	2 (66.7)
60–75	4 (11.8)	3 (8.8)	1 (14.3)	–	–	–		2 (6.9)	1 (25.0)	1 (33.3)

Patient-Reported Advantages of Mobile Applications

The most common patient-reported advantages of mobile application use were the ability to monitor FVIII levels and document infusions 25/138 (18.1%), the ability to choose daily activities according to calculated FVIII levels 25/138 (18.1%) and the ability of physicians to view real-time treatments and symptoms 22/138 (15.9%, Table 1). The distribution of patient's opinion and suggestion is also reported in Supplemental Tables S4 and S5.

Patient Feedback

Patient feedback was highly variable. Many patients provided suggestions for creating more intuitive and user-friendly applications, including reducing the requirement for technical expertise and offering clear instructions. Many also suggested incorporating steps for tracking and monitoring FVIII levels, FVIII stocks, prophylaxis history, medications and alerts for refills or doctor appointments, and simplifying care organization and progress tracking. Other suggestions included adding the ability to view bleed-associated factor levels and improving mobile application compatibility with devices such as smartwatches. Patients also expressed concerns about privacy and security, technical issues such as mobile application malfunctioning after updates and failure to send notifications despite being setup. Overall, patients’ opinions and suggestions provide valuable insights for improving the functionality, usability, and design of the mobile application. The correlation between age group and whether patients provided feedback was not statistically significant (P = .074).

Discussion

This observational, cross-sectional, survey-based study provides real-world data on user experience and satisfaction with mobile healthcare applications for the management of PK-guided interactive hemophilia A prophylaxis. The results of this study describe the demographic and geographical distributions of mobile application management and age-related distribution of documentation management status.

Long-term adherence to prophylactic strategies is crucial for the management of hemophilia A. Previous studies have shown that the use of electronic diaries (eDiaries) or mobile application-based tracking of prophylactic healthcare promotes increased documentation of home-based treatment.^20,21 In the current study, both written paper diaries and mobile applications were the prevalent methods of documenting FVIII levels and tracking prophylactic treatment, with differences observed across participating countries. However, the high rate of written paper diary utilization may not directly represent patient documentation preference, as paper diaries are an obligatory precondition for home care by medical law in certain countries such as Hungary.²² Indeed, despite half of patients in Hungary indicating the use of written paper diaries, it is likely that this proportion was larger than indicated. The use of mobile applications may be assumed to be more readily accepted by younger patients, as the intention to use mobile applications correlates with age and smartphone experience. In this study, however, no significant correlation existed between age group and the type of document management method used. Indeed, consistent use of mobile applications was observed across all age ranges.²³ This may partially be because patients do not independently use mobile applications. Medical personnel from hemophilia centers often assist with downloading and comprehending the application, potentially improving the representation of application use across age groups. No significant correlation was observed between age group and the selection of mobile application type. florio^® HAEMO was the predominant choice across all age ranges, possibly because of its nonproduct-specific nature. The lack of product specificity may allow for the freedom of patient and physician preferences. Indeed, a recent survey in Central Europe revealed an almost 90% satisfaction rating among users, with 87% of those who switched application types indicating that they preferred florio^® HAEMO.²⁴ Furthermore, this study showed that over three-quarters of patients who used florio^® HAEMO felt that they had more certainty in life.

A reasonable proportion of patients adhered to recording the majority of infusions using either a written paper diary or a mobile application. A small proportion of patients reported receiving no instructions to record or how to record infusions, bleeds or swelling/joint pain, possibly reflected further in patient feedback, suggesting that clear instructions and application acceptance by healthcare providers would be valuable. The key aspects of mobile application success are the accessibility and simplicity of applications, as well as their ability to integrate easily into daily life.²⁵ Another key aspect promoting the success of mobile applications is the ease of physician use, as applications provided physician support by ensuring real-time patient information was available. Indeed, the key patient-reported advantages of mobile applications were monitoring FVIII levels, documenting infusions, adapting daily activities to calculated FVIII levels, and their physician's real-time view of symptoms and treatments. These findings indicate the benefits of patient empowerment in controlling personal healthcare and may enhance long-term treatment adherence. Physician engagement with mobile applications was reported by almost a quarter of the patients, indicating that physician acceptance or incorporation of applications into the current healthcare workflow warrants further effort. Similarly, slightly less than one-third of patients reported physician engagement with written paper diaries in Hungary. This is possibly because patients are often required to provide a Microsoft Excel Spreadsheet as a tender requirement of health insurance, which physicians may assist with completing, although unlike patient diaries these spreadsheets are not mandatory by law.

Overall, this study indicated that patients were satisfied with their mobile application experience. Patients prioritized application functionality for tracking FVIII levels, including the visibility of FVIII levels, timely log of infusions, and ability to view future calculated FVIII levels. The ability of a mobile application to connect to a smartwatch is considered the lowest priority. However, patient feedback also indicated that a simplified or more user-friendly method for smartwatch connectivity would be beneficial. Similar issues have been reported for other healthcare applications designed to track bleeding disorders, such as myPROBE, which are currently unable to connect to smartwatches.²⁶ Efforts are thus needed to improve patient experience with mobile applications for managing hemophilia A prophylaxis, particularly as the use of tracking solutions such as eDiaries within populations of patients with hemophilia has been shown to improve the rate and standard of home treatment tracking.^20,21

Limitations

This study has several limitations. First, as this was a cross-sectional, survey-based study, data were collected at a single time point; thus, changes within the population could not be identified. Second, the small sample size of patients enrolled from only five eastern European countries may affect the generalizability of the results and limit their applicability to other regions of the world where healthcare systems, cultural practices, and patient experiences may differ. Third, selection bias may have been affected by the possibility of including specific groups of patients such as those who did not use mobile applications and those who used only one type of mobile application. Since the sample consisted of volunteers who were the first to respond to the survey, and the findings are based on self-reported behaviors, it may not be fully representative of the broader population of individuals with hemophilia A in the examined countries. Furthermore, in countries such as Hungary, the use of paper diaries is mandatory by medical law, which might have led to the underreporting of the use of mobile applications.²² Fourth, the survey questionnaire did not include the option “minority of bleeds were recorded.” This exclusion was intentional to focus on capturing the most accurate and comprehensive data possible for bleeding episodes. Including such an option could have introduced variability and potential inaccuracies in the data collection process. By excluding this option, we aimed to ensure that respondents provided a more definitive account of their bleeding episodes. However, this decision may have limited the granularity of the data collected, and future studies could consider including this option to capture a broader range of patient experiences. Finally, the survey questionnaire used in this study was not validated.

This study also has several important strengths. As a cross-sectional, survey-based study, these results provide real-world evidence of user experience and the advantages of mobile applications for hemophilia A management strategies. Additionally, this study is representative of the patient population, as the number of patients included represents the percentage of patients with hemophilia within each participating country. While this study focuses on patients with hemophilia A, some findings, such as the willingness of older patients to use technology when assisted, could have important implications for other chronic conditions. Future studies could explore the specific ways in which technology can be leveraged to improve management and treatment adherence in populations with chronic disease. It would be valuable to explore how to enhance the role of medical staff in training patients on the use of technology and reinforcing adherence. Additionally, investigating whether digital or paper tracking systems, or a combination of both, best supports patients, alongside the development of educational programs for patient communities, could provide important insights for improving patient care.

Conclusion

In this observational, cross-sectional, survey-based study, no correlation was identified between the methods of prophylaxis management or mobile application type and age range. Most patients who utilized mobile applications were satisfied with their experience and identified the ability to monitor FVIII levels and document infusions, choose daily activities based on calculated FVIII levels and provide physicians with real-time access to symptoms and treatments as the key advantages of using a mobile application. Insightful suggestions provided by patients revealed opportunities to improve application functionality, usability and design, such as through improved medication and FVIII supply tracking. Overall, the findings from this study will aid in the development and customization of patient mobile applications for the unique requirements of patients with hemophilia A.

Supplemental Material

sj-docx-1-jpx-10.1177_23743735251323791 - Supplemental material for Patient Preference and Mobile Application Use in Hemophilia A: A Cross-Sectional Survey from Eastern Europe

Supplemental material, sj-docx-1-jpx-10.1177_23743735251323791 for Patient Preference and Mobile Application Use in Hemophilia A: A Cross-Sectional Survey from Eastern Europe by Laszlo Nemes, Helga Hartmann, Hrvojka Kostelac and Tina Roblek in Journal of Patient Experience

Footnotes

Acknowledgments

The authors thank the patients and members of patient associations who participated in the trial; Hungary: Magyar Hemofília Egyesület; Bulgaria: Bulgarian Haemophilia Association; Romania: Asociația Națională a Hemofilicilor din; Lietuvos žmonių sergančių hemofilija asociacija; Cesky svaz hemofiliku. Data acquisition and statistical analysis assistance were provided by Clinres Farmacija d.o.o. and was compensated by Takeda Pharmaceuticals for the services. Writing, editorial support, and formatting assistance were provided by Rebecca Watkin, PhD, of Cactus Life Sciences (part of Cactus Communications), which was contracted and compensated by Takeda Pharmaceuticals for these services. Funding was provided by Takeda Pharmaceutical Kft, Hungary.

Authors’ Contributions

All authors were involved in drafting and critically revising the manuscript for important intellectual content. All authors have approved the final version of the manuscript for publication. Study concept, design, and protocol development were performed by LN, HH, HK, and TR. Data acquisition was performed by LN, HH, HK, and TR with support from Clinres Farmacija d.o.o. Data analysis and interpretation were performed by LN, HH, HK, and TR with support from Clinres Farmacija d.o.o.

Data Availability

The datasets, including the redacted study protocol, redacted statistical analysis plan, and individual patient data supporting the results reported in this article will be made available within 3 months from the initial request to researchers who provide a methodologically sound proposal. The data will be provided after deidentification in compliance with the applicable privacy laws, data protection regulations, and requirements for consent and anonymization.

Declaration of Conflicting Interests

The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: LN received funding for protocol development and review; is a consultant for CSL Behring, Novo Nordisk, Sobi, and Takeda and a speaker for CSL Behring, Novo Nordisk, Octapharma, Sobi, and Takeda. HH, HK, and TR are employees of Takeda. Writing, editorial support, and formatting assistance were provided by Rebecca Watkin, PhD, of Cactus Life Sciences (part of Cactus Communications), which was contracted and compensated by Takeda Pharmaceuticals for these services.

Ethical Approval

This study was conducted in accordance with the applicable local regulations and ethical and legal requirements of each participating country. Ethical approval for this study was sought from the appropriate Ethics Committees and regulatory bodies in Bulgaria (Bulgarian Drug Agency), Czechia (SÚKL, Clinical Trials of Pharmaceuticals Unit, Clinical Trials of Medicinal Products Department), Hungary (Directorate General of Drug Registration and Methodology, Division of Clinical Trials on National Institute of Pharmacy and Nutrition – OGYÉI), Lithuania (Lithuanian Bioethics Committee), and Romania (National Agency for Medicines and Medical Devices). Each body determined that ethical approval was not required for this observational, survey-based research. This study complied with the International Society for Pharmacoepidemiology Guidelines for Good Pharmacoepidemiology Practices.

Funding

The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: Takeda Pharma Kft., Népfürdő utca 22, 1138 Budapest, Hungary.

Informed Consent

Written informed patient consent/assent was obtained from all participants upon enrolment in compliance with the General Data Protection Regulation (GDPR). Patients could withdraw consent to participate at any point throughout the study and data collection. Informed consent was obtained from all participants involved in the study. For participants under the age of 18, consent was obtained from their parents or legal guardians.

Statement of Human and Animal Rights

This research was conducted in accordance with the ethical standards of the responsible committee on human and animal experimentation, the Helsinki Declaration of 1975 (revised in 2000), and all applicable local regulations and legal requirements of each participating country.

ORCID iD

Tina Roblek

Supplemental Material

Supplemental material for this article is available online.

References

Stonebraker

Bolton-Maggs

PHB

Brooker

, et al. The World Federation of Hemophilia annual global survey 1999–2018. Haemophilia. 2020;26(4):591-600.

Berntorp

Fischer

Hart

, et al. Haemophilia. Nat Rev Dis Primers. 2021;7(1):45.

Centers for Disease Control and Prevention. Data & Statistics on Hemophilia. https://www.cdc.gov/ncbddd/hemophilia/data.html. Accessed November 6, 2023.

Rayment

Chalmers

Forsyth

, et al. Guidelines on the use of prophylactic factor replacement for children and adults with haemophilia A and B. Br J Haematol. 2020;190(5):684-95.

Srivastava

Brewer

Mauser-Bunschoten

, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1-47. doi:10.1111/j.1365-2516.2012.02909.x

Fijnvandraat

Peters

ten Cate

. Inter-individual variation in half-life of infused recombinant factor VIII is related to pre-infusion von Willebrand factor antigen levels. Br J Haematol. 1995;91(2):474-6.

Chen

, et al. Pharmacokinetic studies of factor VIII in Chinese boys with severe hemophilia A: a single-center study. Chin Med J (Engl). 2018;131(15):1780-5.

Kepa

Horvath

Reitter-Pfoertner

, et al. Parameters influencing FVIII pharmacokinetics in patients with severe and moderate haemophilia A. Haemophilia. 2015;21(3):343-50.

World Federation of Hemophilia. WFH guidelines for the management of hemophilia. 3rd ed. https://www1.wfh.org/publications/files/pdf-1863.pdf. Accessed November 6, 2023.

10.

Björkman

. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010;16(4):597-605.

11.

Kahir

Holland

. Managing hemophilia: the role of mobile technology. Smart Homecare Technol Telehealth. 2014;2:39-44. doi:10.2147/SHTT.S40961

12.

Qudah

Luetsch

. The influence of mobile health applications on patient-healthcare provider relationships: a systematic, narrative review. Patient Educ Couns. 2019;102(6):1080-9.

13.

Takeda. myPKFiT. About myPKFiT^®. https://www.advate.com/mypkfit. Accessed November 6, 2023.

14.

McMaster University. WAPPS-Hemo. https://www.wapps-hemo.org/. Accessed November 6, 2023.

15.

Florio. About florio^® HAEMO. https://florio-haemo.com/. Accessed November 6, 2023.

16.

McEneny-King

Foster

Iorio

Edginton

. Data analysis protocol for the development and evaluation of population pharmacokinetic models for incorporation into the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo). JMIR Res Protoc. 2016;5(4):e6559. doi:10.2196/resprot.6559

17.

Narrillos-Moraza

Gómez-Martínez-Sagrera

Amor-García

, et al. Mobile apps for hematological conditions: review and content analysis using the mobile app rating scale. JMIR mHealth uHealth. 2022;10(2):e32826.

18.

Hatem

Long

Best

, et al. Mobile apps for people with rare diseases: review and quality assessment using mobile app rating scale. J Med Internet Res. 2022;24(7):e36691.

19.

Dirzu

Hotea

Jitaru

, et al. Mobile health technology for the personalized therapy of hemophilia. Front Med (Lausanne). 2021;8:711973.

20.

Banchev

Goldmann

Marquardt

, et al. Impact of telemedicine tools on record keeping and compliance in haemophilia care. Hamostaseologie. 2019;39(4):347-54.

21.

Tiede

Bonanad

Santamaria

, et al. Quality of electronic treatment records and adherence to prophylaxis in haemophilia and von Willebrand disease: systematic assessments from an electronic diary. Haemophilia. 2020;26(6):999-1008.

22.

EüM decree on the controlled home treatment of hereditary hemophiliacs. https://net.jogtar.hu/jogszabaly?docid=99800024.eum. Accessed December 14, 2023.

23.

Nunes

Limpo

Castro

. Acceptance of mobile health applications: examining key determinants and moderators. Front Psychol. 2019;10:2791.

24.

Zapotocka

Batorova

Bilic

, et al. First experience of a hemophilia monitoring platform: florio HAEMO. Res Pract Thromb Haemost. 2022;6(2):e12685.

25.

Salisbury

Thomas

O'Cathain

, et al. TElehealth in CHronic disease: mixed-methods study to develop the TECH conceptual model for intervention design and evaluation. BMJ Open. 2015;5(2):e006448.

26.

Germini

Borg Debono

Page

, et al. User-centered development and testing of the online patient-reported outcomes, burdens, and experiences (PROBE) survey and the myPROBE app and integration with the Canadian bleeding disorder registry: mixed methods study. JMIR Hum Factors. 2022;9(1):e30797.

Supplementary Material

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