Skinner M. W., Kucher A., Clearfield E., Curtis R. G., Germini F., Iorio A., Nichol M., Page D., Stonebraker J. Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study. (2025), Book of Abstracts. Haemophilia, 31: 21-109. https://doi.org/10.1111/hae.70032 Objective There is limited comparative quality-of-life (QoL) data… Continue reading Comparing Health Outcomes for Women and Girls with Hemophilia and No Bleeding Disorder – Insights from the Patient Reported Outcomes Burdens and Experiences (PROBE) Study
Author: Alexandra Kucher
Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study
Kucher A, Skinner M. W., Clearfield E., Brennan F., Rotellini D., Colle Y., Wilton P. A. Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study. (2025), Book of Abstracts. Haemophilia, 31: 21-109. https://doi.org/10.1111/hae.70032 Objective The Patient Reported Outcomes, Burdens, and Experiences (PROBE) survey was first developed… Continue reading Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study
International Women’s Day 2025 – Time for Action!
On the 50th Anniversary of International Women’s Day, mark this celebration with us by participating in the PROBE Study. Contributing to this study will help us to accelerate action and break the silence for women with bleeding disorders! Let’s take a stand for the women and girls who have been overlooked in bleeding disorder research… Continue reading International Women’s Day 2025 – Time for Action!
Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial
Skinner M, Chen E, Ibrahim Q, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial. (Abstract). NHF (2023). Objective The Patient Reported… Continue reading Gene Therapy in Hemophilia A: the Impact of Valoctocogene Roxaparvovec on Patient Outcomes – Initial Results from Patient Reported Outcomes, Burdens and Experiences (PROBE) from the GENEr8-1 Trial
Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Skinner M, Chen E, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE. (Abstract). NHF (2023). Introduction People with severe haemophilia A (PwSHA) commonly present with intramuscular bleeding and hemarthrosis, leading to acute and chronic… Continue reading Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Skinner M, Chen E, Kucher A, Germini F, Karimi M, Clearfield E , O’Mahony B , Jain M. Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE. (2023), CCS_Book of abstracts. Haemophilia, 29: 3-53. https://doi.org/10.1111/hae.14781 Introduction People with severe haemophilia A (PwSHA) commonly present with intramuscular bleeding and hemarthrosis,… Continue reading Initial results of the impact of valoctocogene roxaparvovec on pain occurrence and interference: Insights from PROBE
Test-Retest Reliability of a Mobile Application of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study
Curtis R, Wu J, Iorio A, Frick N, Nichol M, Noone D, O’Mahony B, Page D, Stonebraker J, Kucher A, Clearfield E, Skinner MW, Germini F. Test-retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study. Haemophilia. 2024 May;30(3):702-708. doi: 10.1111/hae.14969. Epub 2024 Mar 4. PMID: 38439137. Introduction The… Continue reading Test-Retest Reliability of a Mobile Application of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study
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… Continue reading Self-reflections, lessons learned and suggestions for data quality assurance from a retrospective data analysis from the Canadian Bleeding Disorders Registry
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.… Continue reading PROBE: Brazil data
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… Continue reading Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry
