A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt

NCT04106323 | Unknown DMC

• 1 other identifier: SKhaled20
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region. This study aims to

1. 1.To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region.
2. 2.To investigate modifiable risk factors associated with development of inhibitors in both diseases.
3. 3.To correlate the level of inhibitor with the clinical presentation of the patients.
4. 4.To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.

Conditions

Hemophilia; Von Willebrand Diseases; Christmas Disease

Keywords

Upper Egypt; Factor; Inhibitor

Outcome Measures

Primary Outcomes (3)

• Number of patients with inhibitors

  Frequency of inhibitors among patients with hemophilia A and B, and VWD

  4-days

• Number of patients on demand replacement therapy

  Identification of the relationship between on demand replacement therapy and development of factor inhibitors in the study patients

  7-days

• Number of participants with low or high responding inhibitors

  This would be assessed by of the relationship between inhibitor level and severity of clinical presentation of the patient

  7-days

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients with hemophilia A, B, and Von willebrand's disease who will be admitted at the clinical hematology unit ( Department of Internal Medicine), Assiut University Hospital over one year will be included in the study.

You may qualify if:

• Patients diagnosed congenital hemophilia A, Christmas disease, and VWD

You may not qualify if:

• Patients diagnosed with acquired hemophilia
• Patients below 18 years
• Patients with other bleeding tendencies

Sponsors & Collaborators

• Safaa AA Khaled: lead

Related Publications (11)

• Witmer C, Young G. Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol. 2013 Feb;4(1):59-72. doi: 10.1177/2040620712464509.

  PMID: 23610614 BACKGROUND

• Ghosh K, Shukla R. Future of Haemophilia Research in India. Indian J Hematol Blood Transfus. 2017 Dec;33(4):451-452. doi: 10.1007/s12288-017-0862-4. Epub 2017 Aug 21. No abstract available.

  PMID: 29075053 BACKGROUND

• Schep SJ, Boes M, Schutgens REG, van Vulpen LFD. An update on the 'danger theory' in inhibitor development in hemophilia A. Expert Rev Hematol. 2019 May;12(5):335-344. doi: 10.1080/17474086.2019.1604213. Epub 2019 Apr 25.

  PMID: 30951401 BACKGROUND

• Hay CR, Palmer B, Chalmers E, Liesner R, Maclean R, Rangarajan S, Williams M, Collins PW; United Kingdom Haemophilia Centre Doctors' Organisation (UKHCDO). Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United Kingdom. Blood. 2011 Jun 9;117(23):6367-70. doi: 10.1182/blood-2010-09-308668. Epub 2011 Apr 6.

  PMID: 21471523 BACKGROUND

• Hay CR, DiMichele DM; International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012 Feb 9;119(6):1335-44. doi: 10.1182/blood-2011-08-369132. Epub 2011 Nov 18.

  PMID: 22101900 BACKGROUND

• Gouw SC, van den Berg HM, Oldenburg J, Astermark J, de Groot PG, Margaglione M, Thompson AR, van Heerde W, Boekhorst J, Miller CH, le Cessie S, van der Bom JG. F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis. Blood. 2012 Mar 22;119(12):2922-34. doi: 10.1182/blood-2011-09-379453. Epub 2012 Jan 26.

  PMID: 22282501 BACKGROUND

• Peyvandi F, Mannucci PM, Garagiola I, El-Beshlawy A, Elalfy M, Ramanan V, Eshghi P, Hanagavadi S, Varadarajan R, Karimi M, Manglani MV, Ross C, Young G, Seth T, Apte S, Nayak DM, Santagostino E, Mancuso ME, Sandoval Gonzalez AC, Mahlangu JN, Bonanad Boix S, Cerqueira M, Ewing NP, Male C, Owaidah T, Soto Arellano V, Kobrinsky NL, Majumdar S, Perez Garrido R, Sachdeva A, Simpson M, Thomas M, Zanon E, Antmen B, Kavakli K, Manco-Johnson MJ, Martinez M, Marzouka E, Mazzucconi MG, Neme D, Palomo Bravo A, Paredes Aguilera R, Prezotti A, Schmitt K, Wicklund BM, Zulfikar B, Rosendaal FR. A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A. N Engl J Med. 2016 May 26;374(21):2054-64. doi: 10.1056/NEJMoa1516437.

  PMID: 27223147 BACKGROUND

• Miller CH, Platt SJ, Rice AS, Kelly F, Soucie JM; Hemophilia Inhibitor Research Study Investigators. Validation of Nijmegen-Bethesda assay modifications to allow inhibitor measurement during replacement therapy and facilitate inhibitor surveillance. J Thromb Haemost. 2012 Jun;10(6):1055-61. doi: 10.1111/j.1538-7836.2012.04705.x.

  PMID: 22435927 BACKGROUND

• Soucie JM, Miller CH, Kelly FM, Payne AB, Creary M, Bockenstedt PL, Kempton CL, Manco-Johnson MJ, Neff AT; Haemophilia Inhibitor Research Study Investigators. A study of prospective surveillance for inhibitors among persons with haemophilia in the United States. Haemophilia. 2014 Mar;20(2):230-7. doi: 10.1111/hae.12302. Epub 2013 Nov 22.

  PMID: 24261612 BACKGROUND

• Duncan E, Collecutt M, Street A. Nijmegen-Bethesda assay to measure factor VIII inhibitors. Methods Mol Biol. 2013;992:321-33. doi: 10.1007/978-1-62703-339-8_24.

  PMID: 23546724 BACKGROUND

• Pokras SM, Petrilla AA, Weatherall J, Lee WC. The economics of inpatient on-demand treatment for haemophilia with high-responding inhibitors: a US retrospective data analysis. Haemophilia. 2012 Mar;18(2):284-90. doi: 10.1111/j.1365-2516.2011.02623.x. Epub 2011 Aug 4.

  PMID: 21812862 BACKGROUND

MeSH Terms

Conditions

Hemophilia A; von Willebrand Diseases; Hemophilia B

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; Blood Platelet Disorders; Genetic Diseases, X-Linked

Study Officials

• Howaida A. Nafady, Prof.

  Assiut University Hospial

  STUDY DIRECTOR

Central Study Contacts

Safaa A Khaled, Ass. Prof.

CONTACT

01064170058; safaakhaled2003@gmail.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: September 19, 2019
• First Posted: September 27, 2019
• Study Start: May 10, 2020
• Primary Completion: November 10, 2020
• Study Completion: August 20, 2021
• Last Updated: March 12, 2020

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share