Deeper insight into the misrouted immune response to ADAMTS13 in acquired TTP - a prerequisite for better patient management.
Research Aims of this Study:
"Acquired thrombotic thrombocytopenic purpura (TTP) is the consequence of autoantibodies to ADAMTS13, in remission these autoantibodies are not detectable or kept in check and reappear in relapse. The aim of the proposed studies is to further characterize the autoantibodies to ADAMTS13 and their evolution over time in TTP patients."
How did Dr. J. A. Kremer Hovinga become interested in TTP research:
"Coagulation and pedigrees have fascinated me since I was about 13 or 14 years old. After medical school I worked first as a research fellow and then trained in clinical hematology at the Department of Hematology, Inselspital, Bern University Hospital, Bern, Switzerland. My mentors were Bernhard Lämmle and Miha Furlan, who in the early 1990ies embarked to identify the Von Willebrand factor multimer size regulator, the Von Willebrand factor-cleaving protease, now ADAMTS13, and in 1997 established the link between a severe deficiency of this protease and TTP. I was fortunate to witness this very closely and my fascination for this field has lasted ever since."
How Dr. J. A. Kremer Hovinga believes this research will impact individuals living with or impacted by TTP:
"At present TTP treatment is rather general and unspecific and consists of plasma exchange and immune suppressive agents, such as steroids and rituximab. Characterization and understandingthe autoimmune response to ADAMTS13 is a prerequisite to develop specific treatment, i.e. aiming at the specific elimination of anti-ADAMTS13 antibody producing B-cells (instead of all B-cells as is done by rituximab) or the neutralization of circulating anti-ADAMTS13 antibodies. An anti-ADAMTS13 specific treatment at the appropriate time point is the ultimate goal of the proposed studies."
Comments from Dr. J. A. Kremer Hovinga to donors:
"Rare and ultra-rare diseases, like TTP have been neglected by governmental funding bodies for a long time. Moreover, affected patients, their families and friends have often been quite lonely with their sorrows and fears. Thanks to the commitment of foundations such as Answering T.T.P. this is changing slowly: these rare diseases become visible, patients and researcher are brought together to gain deeper understanding of these diseases and the needs of patients, to develop better treatment for future patients, and the platforms provide a “home” for single patients who suffer from the same rare disease.
The individual stories, the commitment of affected patients, their families and friends supporting Answering T.T.P are instrumental for this change and have given TTP a face far beyond the closed community of experts in the field. "