Musculoskeletal Function in Hemophilia
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|ClinicalTrials.gov 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
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|
|Study Type :||Observational|
|Enrollment :||250 participants|
|Observational Model:||Defined Population|
|Observational Model:||Natural History|
|Official Title:||Musculoskeletal Function in Hemophilia in Developing Countries|
|Study Start Date :||June 2004|
|Study Completion Date :||June 2009|
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 ClinicalTrials.gov identifier (NCT number): NCT00324493
|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|
|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|
|Prof. Glynn Wessels|
|Tygerberg, South Africa|
|Prof. Ampaiwan Chuansumrit|
|Norma De Bosch|
|Principal Investigator:||Alok Srivastava, MD||Christian Medical College, Vellore, India|