Comparison of Efficacy, Safety and Costs of Recombinant FVIII Products Between On-demand and Secondary Prophylaxis Groups in Haemophilia A Patients

Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis

The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII < 1%).

It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment.

The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.

Study Overview

• Status: Completed
• Conditions: Hemophilia
• Intervention / Treatment: Other: Recombinant Factor VIII (Kogenate, BAY14-2222)

• Study Type: Observational
• Enrollment (Actual): 73

Contacts and Locations

Study Locations

• Turkey
  • Multiple Locations, Turkey

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Adult haemophilia patients already on treatment with commercially available recombinant FVIII products.

Description

Inclusion Criteria:

• Age ≥ 18 years
• Severe hemophilia A (FVIII<1%) diagnosis
• Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements
• Previously treated patients with at least >50 exposure days
• Written informed consent signed by patient/legal representative

Exclusion Criteria:

• Currently on immune tolerance treatment
• Platelet count < 75,000/mm3
• Participation in another study
• Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer < 0.6 BU/ml)
• Existence of inhibitor history in family members who also are diagnosed with hemophilia A
• Having been on primary prophylaxis as defined in the introduction
• Participation in another study

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group 1	Other: Recombinant Factor VIII (Kogenate, BAY14-2222); All dosage, frequency and duration for drugs will be under the decision of the treating physician.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group	After 4 years
Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group	After 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of overall bleeding episodes		After 4 years
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)		After 4 years
Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)		After 4 years
Cost-effectiveness (cost of additional joint bleed)		After 4 years
Cost-utility	The costs of care of subjects with haemophilia will be evaluated adopting the perspective of the payer, i.e. the Social Security Institution [SSI].	After 4 years
Comparison of patient compliance between prophylaxis and on-demand therapy groups		After 4 years
Number of spontaneous bleeds		After 4 years
Quality of life as measured with the SF-36		Baseline and after 4 years
Quality of life as measured with Hemo-QoL		Baseline and after 4 years

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

Helpful Links

• Click here to find results for studies related to Bayer products.

Study record dates

Study Major Dates

• Study Start (Actual): January 4, 2013
• Primary Completion (Actual): July 16, 2018
• Study Completion (Actual): May 23, 2019

Study Registration Dates

• First Submitted: March 12, 2013
• First Submitted That Met QC Criteria: March 21, 2013
• First Posted (Estimate): March 26, 2013

Study Record Updates

• Last Update Posted (Actual): May 27, 2020
• Last Update Submitted That Met QC Criteria: May 22, 2020
• Last Verified: May 1, 2020

More Information

Terms related to this study

• Keywords: Observational; Adult; Turkey; Hemophilia
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 16368; KG1210TR (Other Identifier: Company Internal)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No