Musculoskeletal Function in Hemophilia

NCT00324493 | Unknown

• 1 other identifier: MUSFIH2004
• Study Type: observational
• Target: 250
• Locations: 9 countries
• Sites: 10

Brief Summary

Hemophilia, which results from deficiency of factor VIII or IX, is a common hereditary X-linked bleeding disorder affecting up to 10/100,000 population. About 60-70% of them have severe disease (factor level \<1%). This group is characterized by the occurrence of frequent spontaneous bleeding into joints and soft tissues. If inadequately treated, it results in progressive damage to joints and muscles leading to crippling deformities. Close clinical observation of these patients over many years has shown that those with \>1% levels have much less bleeding compared to those with less than 1%. This observation has gained immense clinical importance in planning therapy for these patients. To prevent progressive joint damage, the missing factor needs to be replaced. Much has evolved in this practice in the last 50 years. From administration of whole blood in the beginning, to plasma and cryoprecipitate, to purified plasma-derived concentrates and finally recombinant factor concentrates. The standard of therapy now is to replace factors frequently enough to maintain \>1% factor levels at all times ("prophylaxis") or administer immediately on premonition or earliest signs of bleeding ("on demand" therapy). This has greatly enhanced the quality of life of people with hemophilia. However, the optimal regimens of factor replacement remain to be defined. The definition of what is optimal management of this chronic condition, currently incurable for the vast majority of patients, varies significantly in different parts of the world, depending on practicality and social expectations. Models have care have been developed in Western countries based on careful documentation of outcome over many years. Such data is lacking from developing countries. This multi-center study aims to systematically record the outcome of musculoskeletal function in people with hemophilia in developing countries for the first time and provide information that can help plan care for the 80% of all hemophiliacs in the world who live in these countries. Currently there is no well documented model of care at the range of factor replacement practiced in these countries nor is there any significant information on the long-term outcome of musculo-skeletal function among these patients.

Conditions

Hemophilia

Keywords

Severe Hemophilia; Musculoskeletal Function; Factor Replacement

Eligibility Criteria

• Age: 5 Years - 15 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Severe hemophilia, defined as factor assay showing \<1% activity (assay to be done using standard reagents), between 5-15 years of age
• Be willing to come for evaluation at least once in 6-12 months for 5 years

You may not qualify if:

• Detectable inhibitors by screening tests at recruitment

Sponsors & Collaborators

• Christian Medical College, Vellore, India: lead

Study Sites (10)

Raul Perez Bianco

Buenos Aires, Buenos Aires F.D., Argentina

Location

Margareth Castro Ozelo /

Chagas, Cidade Univrsitaria Zeferino Vaz-Campinas-Sp, 13 083 970, Brazil

Location

Elbio A.D' Amico / Jorge

São Paulo, São Paulo, 01246 903, Brazil

Location

Magdy EI Ekiaby

Cairo, Cairo Governorate, Egypt

Location

Christian Medical College

Vellore, Tamil Nadu, 632004, India

Location

Mohammad Reza Baghaipour

Tehran, Tehran Province, 14158 63675, Iran

Location

Tien Sim Leng

Singapore, 169608, Singapore

Location

Prof. Glynn Wessels

Tygerberg, South Africa

Location

Prof. Ampaiwan Chuansumrit

Bangkok, Thailand

Location

Norma De Bosch

Caracas, Venezuela

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Alok Srivastava, MD

  Christian Medical College, Vellore, India

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: DEFINED POPULATION
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: May 9, 2006
• First Posted: May 11, 2006
• Study Start: June 1, 2004
• Study Completion: June 1, 2009
• Last Updated: November 30, 2006

Record last verified: 2005-06

Locations

AR (1); BR (2); TH (1); IR (1); SG (1); EG (1); IN (1); VE (1); ZA (1)