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Musculoskeletal Function in Hemophilia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00324493
Recruitment Status : Unknown
Verified June 2005 by Christian Medical College, Vellore, India.
Recruitment status was:  Active, not recruiting
First Posted : May 11, 2006
Last Update Posted : November 30, 2006
Information provided by:
Christian Medical College, Vellore, India

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.

Condition or disease

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Study Type : Observational
Enrollment : 250 participants
Observational Model: Defined Population
Observational Model: Natural History
Time Perspective: Longitudinal
Time Perspective: Prospective
Official Title: Musculoskeletal Function in Hemophilia in Developing Countries
Study Start Date : June 2004
Study Completion Date : June 2009

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Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Detectable inhibitors by screening tests at recruitment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00324493

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Raul Perez Bianco
Buenos Aires, Ciudad de Buenos Aires, Argentina
Margareth Castro Ozelo /
Chagas, Cidade Univrsitaria Zeferino Vaz-Campinas-Sp, Brazil, 13 083 970
Elbio A.D' Amico / Jorge
Sao Paulo, Brazil, 01246 903
Magdy EI Ekiaby
Cairo, Egypt
Christian Medical College
Vellore, Tamil Nadu, India, 632004
Iran, Islamic Republic of
Mohammad Reza Baghaipour
Teheran, Iran, Islamic Republic of, 14158 63675
Tien Sim Leng
Singapore, Singapore, 169608
South Africa
Prof. Glynn Wessels
Tygerberg, South Africa
Prof. Ampaiwan Chuansumrit
Bangkok, Thailand
Norma De Bosch
Caracas, Venezuela
Sponsors and Collaborators
Christian Medical College, Vellore, India
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Principal Investigator: Alok Srivastava, MD Christian Medical College, Vellore, India
Layout table for additonal information Identifier: NCT00324493    
Other Study ID Numbers: MUSFIH2004
First Posted: May 11, 2006    Key Record Dates
Last Update Posted: November 30, 2006
Last Verified: June 2005
Keywords provided by Christian Medical College, Vellore, India:
Severe Hemophilia
Musculoskeletal Function
Factor Replacement
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn