Self-reflections, lessons learned and suggestions for data quality assurance from a retrospective data analysis from the Canadian Bleeding Disorders Registry

Iorio A, Keepanasseril A, Ibrahim Q, Iserman E, Blaser H. Self-reflections, lessons learned and suggestions for data quality assurance from a retrospective data analysis from the Canadian Bleeding Disorders Registry. (2024), POSTER ABSTRACT (PP-026). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

Real-world evidence (RWE) is used to complement primary evidence from clinical trials on safety and efficacy and to generate clinical effectiveness data. Theoretically, RWE is not subject to ‘on trial effect’, and it shows the true impact of intervention in unselected populations. Barriers to generating RWE are mostly stemming from the incomplete availability of relevant data points in the real-world setting. Aim: Analyse strengths and limitations of the ‘Real-World Effectiveness of PEGylated, recombinant Antihaemophilic Factor (Rurioctocog alfa pegol) Prophylaxis in Patients with Haemophilia A in Canada: A Retrospective, Intra-Patient Comparison with a Before-AfterDesign’.

Methods

A retrospective study, before-after design, comparing PK, clinical and PR outcomes for patient switched from SHL-FVIII or EHL-FVIII products to Rurioctocog alfa pegol. Outcomes: individual PK parameters (half-life, time spent above factor levels of0.01, 0.03 and 0.05 IU/mL), factor utilisation, bleed rates, QoL (PROBE)and joint function (HJHS). Pre-specified subgroups: previous treatment with SHL-FVIII versus EHL-FVIII, history of inhibitors versus no inhibitors, and age ≤12 versus >12 years. Strengths and limitations of the approach, including the amount/quality of data available for the analysis of the different outcomes, were analysed.

Results

Ninety-eight severe haemophilia A inhibitor-free patients qualified for the analysis. Thirty-seven patients had available PK data. There were four patients with history of inhibitors, six <12 years and eight patients formerly treated with SHL; four had HJHS and four had PROBE data. Clinical, PK and utilisation results have been presented elsewhere.

Conclusions

PROBE data shows a good cross-section of people with Haemophilia A, B and carriers. Brazil has free Selection bias is anticipated to be lower in retrospective design versus post-marketing surveillance (PMS) studies, which also threaten national registries data integrity. Included (n = 37) and excluded (n = 61) patients were similar in terms of age distributions, BMI, and number of surgery or traumatic bleedings, confirming that the retrospective analysis did not identify a selected population. Lim-ited availability of HJHS and PROBE data suggests that routine clinical practice does not yet include standardised recording of patient centred/reported outcomes, hampering the measurement of health care interventions’ value. There is a significant opportunity to improve the breadth/quality of data generation in the future to demonstrate the true impact of factor replacement in routine clinical practice.

Disclosures

Alfonso Iorio: Bayer, CSL, Pfizer, Roche, Sanofi/Sobi, Takeda; Heiko Blaser: Takeda.

View Poster: PP_26_WFH2024

PROBE: Brazil data

Pietrobelli T, Skinner M. PROBE: Brazil data. (2024), POSTER ABSTRACT (PP-165). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

The Brazilian Haemophilia Federation (FBH) carried out a campaign to increase responses to the PROBE (Patient Reported Outcomes, Burdens, and Experiences) questionnaire, which collaborates with the World Haemophilia Federation and was integrated into myWBDR and GTR. To have robust data on the quality of life, treatment and daily life of people with Haemophilia A and B. The data collected is intended to be used in FBH advocacy actions, to improve Public Policies in Brazil.

Methods

PROBE has real-life data from patients, which is why FBH has been a partner in the study since 2018 and has a specific response link that allows you to know the state where the person lives and the distance from their home to the HTC. The information collected by PROBE allows FBH to hold meetings with government authorities and propose improvements for the treatment of people with haemophilia in all 27 federative units in Brazil.

Results

PROBE has 679 contributions to the questionnaire in Brazil. With 321 people with Haemophilia A, 49 with Haemophilia B, 35 carriers and 274 people without coagulopathy, this last group is considered a comparative group for quality of life. Considering 405 people with Haemophilia A, B and carriers, 168 have Severe Haemophilia and 45 Moderate. Around 65% undergo treatment at home and 28% go tothe HTC for treatment. 75% perform 2–3 infusions per week and only 2.25% infuse daily. 41.75% do not have chronic pain and 58.25% admit to living with chronic pain, of which 85% have chronic pain in the target joints.

Conclusions

PROBE data shows a good cross-section of people with Haemophilia A, B and carriers. Brazil has free treatment through the Public Health System, which is an example for the world and, even so, there are windows of improvement that should be taken advantage of. With this data, FBH will work to improve Public Policies with government authorities to improve access to treatment and quality of life.

Disclosures

Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics.

View Poster: PP_165_WFH2024

Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry

Naccache M, Konkle B, Peyvandi F, Miesbach W, O’Mahony B, Pile S, Youttananukorn T, Coffin D, Pierce G. Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (MP-021). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

The World Federation of Haemophilia (WFH) launched the Gene Therapy Registry (GTR) aimed at gathering comprehensive data on all people with haemophilia (PWH) who receive gene therapy worldwide.

Methods

The GTR was designed to standardise and centralise global data collection for the gathering and dissemination of gene therapy data. The GTR is a prospective, observational, and longitudinal registry. Data entry occurs once, either directly from haemophilia treatment centre (HTC) into the GTR or through data transfer from National Registries. The GTR Scientific Advisory Board oversees all data entered into the GTR and its dissemination. Specificde-identified data will be shared with various stakeholders: participating patients will have access to view their own data; HTCs and National Registries will receive aggregated global safety data; industry part-ners will receive product-specific data, and regulatory agencies and health technology assessment organisations can request specific data to inform their decisions.

Results

The WFH is engaged with a broad network of collabora-tors, and the GTR National Registries & HTC Consortium has been established to foster dialogue, obtain feedback from our collaborators, and establish mutually beneficial collaboration. This group includes representatives from Brazil, Ireland, Saudi Arabia and Sweden, and registries from Australia, Canada, France, Germany, Japan, the Netherlands, Spain, the United Kingdom and the United States. The European Medicines Agency (EMA) issued a pivotal letter of support to the WFHGTR. The CHMP endorses the GTR as the worldwide registry for consolidating all international data on PWH who receive gene therapy and encourages collaboration of all HTCs and National Registries, stating that the WFH GTR is of particular value for post approval safety and efficacy studies of gene therapies and recommending its use as a planned data source for mandated Phase IV studies.

Conclusions

The GTR facilitates the accumulation of data in one registry and supports the efficient dissemination of valuable information to all stakeholders, advancing our understanding of gene therapy’s safety, efficacy, and long-term effects. In the current landscape of numerous registries in haemophilia around the world, the success of the GTR depends on collaborative relationships with all stakeholders, including patients, HTCs, National Registries, industry partners and regulatory bodies.

Disclosures

Barbara Konkle: Be Biotherapy, Biomarin, Novo Nordisk, Pfizer, Sanofi; Flora Peyvandi: BioMarin Pharmaceutical Inc., CSL Behring, F. Hoffmann-La Roche Ltd., Grifols, Sanofi, Sobi, Takeda; Wolfgang Miesbach: Bayer, BioMarin, Biotest, Chugai, CSL Behring, Free-line, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/shire; Brian O’Mahony: Biomarin, CSL Behring, Freeline, Irish Haemophilia Society, Pfizer, Roche; Mike Makris: Grifols, Novo Nordisk, Sanofi, Takeda; Steven Pipe: Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Genentech/Roche, GeneVentiv and Equilibra Bioscience, Sanofi, Takeda, Spark Therapeutics, uniQure, Siemens; Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics; Glenn F. Pierce: ASC Therapeutics, Be Bio, BioMarin, Decibel Therapeutics, Frontera, Intellia, Metagenomi, Novo Nordisk, Pfizer, Regeneron, Spark Therapeutics, Third Rock Ventures.

View Poster: MP_21_WFH2024

Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry

Youttananukorn T, Konkle B, Peyvandi F, Naccache M, Miesbach W, O’Mahony B, Makris M, Pipe S, Skinner M, Coffin D, Pierce G.Long-term retention plan through myGTR – a patient engagement tool from World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (PP-054). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

The World Federation of Haemophilia (WFH) Gene Therapy Registry (GTR) is designed to collect comprehensive clinical data on all people with haemophilia (PWH) who receive gene therapy (GT) globally. To complement data from the GTR, the WFH developed myGTR – a patient engagement tool aimed at collecting patient-reported outcome (PRO) data. PRO data are important and are part of shared decision-making process that could lead to meaningful care and treatment.

Methods

To reduce potential data gap after GT and to ensure long-term engagement between haemophilia treatment centre (HTC) and PWH, the GTR is developing a retention plan. The patient engagement plan of the GTR includes myGTR – the foundational element, data visualisation through dashboards, video/podcasts with experts, and a dedicated website with latest news about GT.

Results

Whilst developing myGTR, the WFH held focus groups to discuss which PRO data are important to collect, at which frequency, and how to capture PWH’s experience on GT. The groups indicated mobile app fatigue and requested a simple tool. myGTR is not available at any app store. It was developed as a web-based application. Patients can choose their preferred contact method – email or text message – to access myGTR and provide PRO data via an interactive digital assis-tant. The PRO data are bleeds, treatments and health related quality of life (HR-QOL) including the Patient Reported Outcomes Burdens and Experiences (PROBE), coreHEM Mental Health Outlook (core-HEM MHO) and EQ-5D-5L. At regular interval (4 times during the first year, 2 times per year thereafter), the patients will be prompted to answer two simple questions about their health status since GT infusion, and complete two out of three HR-QOL questionnaires on a rotational basis.

Conclusions

To monitor GT’s safety, efficacy and long-term effects, engagement from both HTCs and patients is critical. The GTR is a platform for HTCs whereas myGTR allows the patients to continue providing data on their health status and HR-QOL in a simple manner. When working hand-in-hand, both tools can improve patient care and treatment outcomes as well as our understanding of benefits and risks of GT.

Disclosures

Barbara Konkle: Be Biotherapy, Biomarin, Novo Nordisk, Pfizer, Sanofi; Flora Peyvandi: BioMarin Pharmaceutical Inc., CSL Behring, F. Hoffmann-La Roche Ltd., Grifols, Sanofi, Sobi, Takeda; Wolfgang Miesbach: Bayer, BioMarin, Biotest, Chugai, CSL Behring, Free-line, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/shire; Brian O’Mahony: Biomarin, CSL Behring, Freeline, Irish Haemophilia Society, Pfizer, Roche; Mike Makris: Grifols, Novo Nordisk, Sanofi, Takeda; Steven Pipe: Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Genentech/Roche, GeneVentiv and Equilibra Bioscience, Sanofi, Takeda, Spark Therapeutics, uniQure, Siemens; Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics; Glenn F. Pierce: ASC Therapeutics, Be Bio, BioMarin, Decibel Therapeutics, Frontera, Intellia, Metagenomi, Novo Nordisk, Pfizer, Regeneron, Spark Therapeutics, Third Rock Ventures.

View Poster: PP_54_WFH2024

Physical functioning and pain in older men with haemophilia

O’Callaghan S, Parikh S, Bishop L, Caris S. Physical functioning and pain in older men with haemophilia. (2024), POSTER ABSTRACT (PP-161). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

It is well recognised that physical functioning can be impaired and pain common as men with haemophilia (MWH) age, due to complications from their bleeding disorder. However, it can be difficult to quantify the difference between their experience and that of men without a bleeding disorder (MNBD)of equivalent age in the general population. The PROBE (Patient Reported Outcomes Burdens Experiences) questionnaire is an internationally validated tool defining the impact of haemophilia on quality of life from the patient perspective. In 2020 the PROBE Australia Study compared the experience of MWH and MNBD, all of whom were aged 45 years and over.

Methods

A total of 106 questionnaire respondents aged 45 years and over (MWH: n = 57; MNBD: n = 49) were recruited via Australian community networks. PROBE questions included use of a mobility aid or assistive device, difficulties with activities of daily liv-ing, acute and chronic pain and use of medications for pain in the last 12 months.

Results

Differences between MWH and MNBD were pronounced. Overall MWH reported that 44% (25/57) used a mobility aid, 54% (31/57) had difficulties with activities of daily living, 58% (33/57) experienced acute pain, 74% (42/57) chronic pain and 79% (45/57) used medication for pain in the last 12 months. This was markedly higher than MNBD: none reported problems with mobility, 6% (3/49) reported problems with activities of daily living, 27% (13/49) had acute pain, 41% (20/49) chronic pain, and 61% (30/49) used medication for pain. A higher proportion of men with moderate or severe haemophilia (n = 28) reported physical function problems or pain: 61% (17/28) needed mobility aids, 79% (22/28) had difficulties with activities of daily living, 79% (22/28) had acute pain, 86% (24/28) chronic pain, and 89% (25/28) had used medi-cation for pain. Men with mild haemophilia (n = 29) also reported problems and pain more often than MNBD, including 28% (8/29) reporting mobility problems and 31% (9/29) with activities of daily living.

Conclusions

Validated haemophilia-specific tools such as PROBE are an important way to quantify the substantial differences in quality of life between older men with haemophilia and men without a bleeding disorder of equivalent age.

View Poster: PP_161_WFH2024

Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: effects on patient-oriented outcomes measured using thePatient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study

Pipe S, Abdelkader W, Clearfield E, Kucher A, Joseph B, Braverman J, Galante N, Monahan P, Ibrahim Q, Iorio A, Germini F, Skinner M. Gene therapy with the Padua variant of a codon-optimized human factor IX gene etranacogene dezaparvovec in people with hemophilia B: effects on patient-oriented outcomes measured using thePatient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire in the HOPE-B study. (2024), POSTER ABSTRACT (PP-164). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

The Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire was used to measure patient-oriented outcomes in the HOPE-B trial. Determine the effect of a single dose of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec) on the quality of life and the burden of the disease as measured by the PROBE questionnaire.

Methods

In this phase 3, open-label, single-arm trial, people with severe to moderately severe haemophilia B, after factor IX prophylaxis for ≥6 months (lead-in period), received one infusion of etranacogene dezaparvovec. The PROBE questionnaire was admin-istered at enrolment, during the lead-in period, and at 6 months, 1, 2and 3 years after the treatment. The PROBE score was calculated and ranged from 0 to 1 (worst to best health status possible). Intra-patientchanges in PROBE scores were analysed using a two-level linear mixed model (within patient repeated observations and random intercepts).

Results

Fifty-four adult males received the treatment, and PROBE data were available for 48 participants. The characteristics of the population and the mean PROBE score at the various time points are reported in Table 1. Using the baseline as a reference, there was an average change (95% confidence interval) in the PROBE score of 0.04 (0.02, 0.07) at 6 month/1 year, which persisted at 2–3 years, 0.04(0.02, 0.06). Nine (22.5%) participants had an improvement of at least0.1 in the PROBE score, five (12.5%) had a worsening of at least 0.1. Changes in the responses to the core PROBE questions are reported in Table 2. At three years, there was a 23.8% (95% CI -41.7, −5.8) reduction in the prevalence of participants having experienced acute pain in the previous 12 months. Participants with at least one target joint at baseline demonstrated a trend towards reduction in the proportion of participants experiencing difficulties with activity of daily life, without reaching the cut off for statistical significance.

Conclusions

Administering a single dose of etranacogene dezaparvovec to patients with hemophilia B led to improvement of mean Probe score maintained through at three years.

Disclosures

Steven Pipe: Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Genentech/Roche, GeneVentiv and Equilibra Bioscience, Sanofi, Takeda, Spark Therapeutics, uniQure, Siemens; Bernard Joseph: CSL Behring; Julia Braverman: CSL Behring; Nicholas Galante: CSL Behring; Paul Monahan: CSL Behring; Alfonso Iorio: Bayer, CSL, Pfizer, Roche, Sanofi/Sobi, Takeda; Federico Germini: Bayer, Biomarin, CSL Behring, Novo Nordisk, Pfizer, Roche, Takeda; Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics

View Poster: PP_164_WFH2024

Summary data from the first collaboration of PROBE and Drustvo Hemofilikov Slovenije (DHS) – an ongoing quality of life study

WFH 2024 – PROBE and Slovenia PosterKucher A, Clearfield E, Kavčič M, Urbančič M, Živić Kavčič M and Skinner M. Summary data from the first collaboration of PROBE and Drustvo Hemofilikov Slovenije (DHS) – an ongoing quality of life study. (2024), POSTER ABSTRACT (PP-162). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

Drustvo Hemofilikov Slovenije (DHS) and the Patient Reported Outcomes Burdens and Experiences (PROBE) study have initiated a collaboration to discover the quality of life (QoL) for people with hemophilia (PWH) in Slovenia.

Methods

Data was collected using PROBE’s web-based questionnaire. PROBE collects data in four categories: personal demographics, general health problems, hemophilia-specific health problems, and the Euro-QoL 5 dimensions 5 levels (EQ-5D-5L). Summary descriptive statistics are provided.  

Results

Twenty-four PWH A and B participated (Table 1); 91.7% (n=22) were PWHA. The average age (SD) was 44.3 (18.4) years. Most participants had severe hemophilia 62.5% (n=15), moderate and mild hemophilia were reported by 16.7% (n=4) and 20.8% (n=5), respectively. Nineteen (79.2%) reported access to prophylaxis treatment, and two (8.3%) reported using on-demand treatment. One person reported that treatment was not available. In the family section, 58.3% (n=14) reported being married/in a long-term relationship and 54.2% (n=13) have children. Frequency of use of pain medication was low: 45.8% (n=11) reported using it “rarely” (1-5% of the time), and 25% (n=6) selected that they did not use any pain medication. The presence of acute pain and chronic pain was reported by 50% (n=12) and 70.8% (n=17), respectively. Fifty percent (n=12) indicated their chronic pain is due to target joint/s. Seventeen people (70.8%) indicated they had a target joint. PROBE and EQ-5D-5L scores range from 0 to 1, with a score closer to 1 meaning better QoL. Mean (SD) scores for PROBE and EQ-5D-5L were 0.803 (0.16) and 0.841 (0.14), respectively.

Conclusions

Collaboration between DHS and PROBE launched recently, with a goal to administer PROBE to at least half of the severe and moderate population of PWH in Slovenia. These data from the first set of participants shows the impact and importance of QoL data collection for the patient organization. PROBE can be used to measure access to the treatment, one of the most valuable indications of positive impact on QoL for PWH. PROBE also measures other key outcomes affecting QoL for PWH. Data collection is ongoing, and further analyses will be completed to understand the impact of living with hemophilia in Slovenia.

View Poster: PROBE and Slovenia Poster

National Member Organization’s PROBE Data Dashboard Update – Adding A Pain Dashboard Page for Better Understanding of the Pain Impact in People with Hemophilia

Kucher A, Clearfield E, Skinner M and PROBE Investigators. National Member Organization’s PROBE Data Dashboard Update – Adding A Pain Dashboard Page for Better Understanding of the Pain Impact in People with Hemophilia. (2024), POSTER ABSTRACT (PP-167). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016

Introduction

The Patient Reported Outcomes, Burdens and Experience (PROBE) study has been implemented in 105+ countries and translated into 50+ languages. How does the study benefit national member organizations (NMO)? The PROBE team has developed an interactive dashboard utilizing data from the pain elements of the PROBE questionnaire, a hemophilia-specific quality of life instrument. This dashboard can be provided upon request to participating NMOs for analysis and study of their country’s data with options to aggregate data, generate graphs, and utilize filters to support and specify data analyses.

Methods

The first development of the PROBE NMO Dashboard started in 2017. The dashboard was developed using Power BI, Microsoft software. Since then, the dashboard has been modified to provide better data picture for NMOs to report to health policy decision makers or for use to test and analyze hypotheses for publications. The PROBE questionnaire asks about the use of pain medication, the presence of acute and chronic pain, when pain occurs, and whether pain causes interference in a person’s life for things such as activity, mood, relationship with others.

Results

The importance of detailed PROBE responses regarding pain has been discussed among the PROBE Investigator team and approved for modification. The newest upgrade to the dashboard is a specific Pain page that covers detailed data about acute and chronic pain interference and occurrences, activities of daily living, the EQ-5D-5L pain dimension, PROBE anxiety and depression items, PROBE score and EQ-5D-5L score.

Conclusions

The pain dashboard demonstrates that a person’s experiences with pain are highly important for reporting on quality of life for people with hemophilia. Having the ability to compare both a control group and hemophilia group with pain occurrences and interferences can support claims of better or worse quality of life that could be addressed through healthcare management or and improved access to treatment. Analyzing PROBE and EQ-5D-5L together demonstrates the importance of using both generic and disease specific quality of life instruments. The pain dashboard will be a valuable asset for NMOs for pain reports and management for people with hemophilia.

View Poster: Pain Dashboard Page

Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study

Curtis R, Wu J, Iorio A, Nichol M, Germini F, Kucher A, Skinner M. Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study. (2023), POSTER ABSTRACTS (PO077). Haemophilia, 29: 24-202. https://doi.org/10.1111/hae.14715

Objective

To investigate the test-retest reliability of the PROBE questionnaire mobile application (MyPROBE).

Methods

People with hemophilia (PWH) including carriers and individuals without a bleeding disorder who attended hemophilia related workshops were invited to participate in this study. Additional participants were recruited through social media. Participants completed PROBE anonymously 3 times: twice on the MyPROBE app (Time 1 and Time 2, ~24 hours apart) and once on the web portal (Time 3, ~14 days after T1). Test-retest reliability was measured calculating the Cohen’s Kappa coefficient for categorical variables, and the correlation coefficient for continuous variables. 

Results

Forty-eight participants were enrolled with a median age (range) of 56 (27-78) years. Of these, 13 (27.1%) were PWH, 12 (25.0%) were carriers of hemophilia A or B and 23 (47.9%) were individuals without a bleeding disorder. On general health domain questions, Kappa coefficients ranged from 0.72 to 1.00, indicating substantial to almost perfect agreement using Cohen’s Kappa for all items (T1 vs T2). T2 vs T3 values ranged from 0.64 to 0.97 with only the acute pain-related questions scoring less than almost perfect agreement. For hemophilia-related domain questions (T1 vs T2), Kappa coefficients ranged from 0.49 to 1.00. Of these, 5 of 8 items were in almost perfect agreement. Values for T2 vs T3 ranged from 0.34 to 1.00, with the time-based bleeding question showing the only coefficient that scored below substantial agreement. For overall health-related quality of life, EQ-5D-5L index scores had a paired mean difference of -0.01 for T1 to T2 and 0.01 for T2 to T3 indicating a near perfect correlation. The correlation coefficients for these two time points were 0.89 and 0.83 respectively. Reliability of the MyPROBE app showed substantial to almost perfect agreement with the web version (T2 to T3). Correlation coefficient of the EQ-visual analog scale (EQ-VAS) for T1 to T2 was 0.90 (0.83 – 0.94) and 0.71 (0.53 – 0.83) for T2 to T3.

Conclusions

The test-retest exercise showed substantial to almost perfect agreement in a majority of questions for the app vs app, and a high correlation for the web vs app. The results suggest the MyPROBE app is a reliable tool to assess patient reported outcomes for PWH and control populations independently of the platform used for its completion.

View Poster: HERE

Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study

Curtis R, Wu J, Iorio A, Nichol M, Germini F, Kucher A, Skinner M. Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study. (2023), POSTER ABSTRACTS (PO077). Haemophilia, 29: 24-202. https://doi.org/10.1111/hae.14715

Objective

To investigate the test-retest reliability of the PROBE questionnaire mobile application (MyPROBE).

Methods

People with hemophilia (PWH) including carriers and individuals without a bleeding disorder who attended hemophilia related workshops were invited to participate in this study. Additional participants were recruited through social media. Participants completed PROBE anonymously 3 times: twice on the MyPROBE app (Time 1 and Time 2, ~24 hours apart) and once on the web portal (Time 3, ~14 days after T1). Test-retest reliability was measured calculating the Cohen’s Kappa coefficient for categorical variables, and the correlation coefficient for continuous variables. 

Results

Forty-eight participants were enrolled with a median age (range) of 56 (27-78) years. Of these, 13 (27.1%) were PWH, 12 (25.0%) were carriers of hemophilia A or B and 23 (47.9%) were individuals without a bleeding disorder. On general health domain questions, Kappa coefficients ranged from 0.72 to 1.00, indicating substantial to almost perfect agreement using Cohen’s Kappa for all items (T1 vs T2). T2 vs T3 values ranged from 0.64 to 0.97 with only the acute pain-related questions scoring less than almost perfect agreement. For hemophilia-related domain questions (T1 vs T2), Kappa coefficients ranged from 0.49 to 1.00. Of these, 5 of 8 items were in almost perfect agreement. Values for T2 vs T3 ranged from 0.34 to 1.00, with the time-based bleeding question showing the only coefficient that scored below substantial agreement. For overall health-related quality of life, EQ-5D-5L index scores had a paired mean difference of -0.01 for T1 to T2 and 0.01 for T2 to T3 indicating a near perfect correlation. The correlation coefficients for these two time points were 0.89 and 0.83 respectively. Reliability of the MyPROBE app showed substantial to almost perfect agreement with the web version (T2 to T3). Correlation coefficient of the EQ-visual analog scale (EQ-VAS) for T1 to T2 was 0.90 (0.83 – 0.94) and 0.71 (0.53 – 0.83) for T2 to T3.

Conclusions

The test-retest exercise showed substantial to almost perfect agreement in a majority of questions for the app vs app, and a high correlation for the web vs app. The results suggest the MyPROBE app is a reliable tool to assess patient reported outcomes for PWH and control populations independently of the platform used for its completion.

View Poster: HERE