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Topic Suggestion Description

View Topic Suggestion Disposition (PDF) 1.2 MB

Date submitted: November 20, 2013

Briefly describe a specific question, or set of related questions, about a health care test or treatment that this program should consider.
As you know marrow transplantation is recomanded in DOCK8 deficiency because this disease is worsening with age and always led to death in the second decad of age, but theses recommandations are based on few clinical trials, In an other hand our case has also systemic lupus eythematosus SLE witch requires immunosupressive medication this association is a challenge for clinicians viewpoint treatment. some papers reports goud results of bone marrow tranplantation in SLE

My Idea is to propose this patient for a trial for bone marrow transplantation in a context of research; it's a challenging case that not every center can manage successfully

Importance

Describe why this topic is important.
DOCK8 mutation has recently been discovered in 2009, research in this domain is Research in this area is still very active in order to characterize the disease and provide therapeutic solutions given its very serious nature . Work on this very particular terrain where infectious diseases, tumoral and autimmune diseases intesect, can open therapeutic horizons in these conditions or even make us reconsider our way to treat patients with multiple diseases

Potential Impact

How will an answer to your research question be used or help inform decisions for you or your group?
Allogeneic hematopoietic cell transplantation is reported as curative in DOCK8 patients but data in this domain are still insufficient. our case witch is concerning a 10 years old represents a challenging case since she also has SLE and antiphospholipid syndrome.

Nominator Information

Other Information About You: (optional)
Please choose a description that best describes your role or perspective: (you may select more than one category if appropriate)
zineb Jouhadi,Professor of pediatrics , infectiuos diseases , medical school of casablanca Morocco
If you are you making a suggestion on behalf of an organization, please state the name of the organization
children's hospital of casablanca Morocco
Please tell us how you heard about the Effective Health Care Program
by internet