Results 31 to 40 of about 79,367 (221)

Development and validation of a predictive model for death in acquired severe ADAMTS13 deficiency-associated idiopathic thrombotic thrombocytopenic purpura: the French TMA Reference Center experience

Haematologica, 2012
Background Acquired thrombotic thrombocytopenic purpura is still associated with a 10–20% death rate. It has still not been possible to clearly identify early prognostic factors of death.
Ygal Benhamou, Cyrielle Assié, Pierre-Yves Boelle, Marc Buffet, Rana Grillberger, Sandrine Malot, Alain Wynckel, Claire Presne, Gabriel Choukroun, Pascale Poullin, François Provôt, Didier Gruson, Mohamed Hamidou, Dominique Bordessoule, Jacques Pourrat, Jean-Paul Mira, Véronique Le Guern, Claire Pouteil-Noble, Cédric Daubin, Philippe Vanhille, Eric Rondeau, Jean-Bernard Palcoux, Christiane Mousson, Cécile Vigneau, Guy Bonmarchand, Bertrand Guidet, Lionel Galicier, Elie Azoulay, Hanspeter Rottensteiner, Agnès Veyradier, Paul Coppo   +30 more
doaj   +1 more source

Thrombotic thrombocytopenic purpura [PDF]

The Journal of the American Osteopathic Association, 1989
Once formerly thought to be a rare disorder, thrombotic thrombocytopenic purpura (TTP) is becoming increasingly recognized. It is characterized by a pentad of clinical findings, including microangiopathic hemolytic anemia, thrombocytopenic purpura, neurologic and renal abnormalities, and fever.
openaire   +3 more sources

Characterization and treatment of congenital thrombotic thrombocytopenic purpura.

Blood, 2019
Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare thrombomicroangiopathy caused by an inherited deficiency of a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13).
F. Alwan, C. Vendramin, R. Liesner, A. Clark, W. Lester, T. Dutt, W. Thomas, R. Gooding, T. Biss, Henry G. Watson, N. Cooper, R. Rayment, T. Cranfield, J. J. Veen, Q. Hill, Sarah Davis, J. Motwani, N. Bhatnagar, Nicole Priddee, M. Dávid, Maeve P. Crowley, J. Alamelu, H. Lyall, J. Westwood, M. Thomas, M. Thomas, M. Scully, M. Scully   +27 more
semanticscholar   +1 more source

Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

Clinical Pharmacology &Therapeutics, EarlyView.
Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami, Kako Shimotsumagari, Yasunari Sadatsuki   +2 more
wiley   +1 more source

CNR2 functional variant (Q63R) influences childhood immune thrombocytopenic purpura

Haematologica, 2011
Immune thrombocytopenic purpura is an acquired autoimmune disorder that is the most common cause of thrombocytopenia in children. The endocannabinoid system is involved in immune regulation.
Francesca Rossi, Silvia Mancusi, Giulia Bellini, Domenico Roberti, Francesca Punzo, Simona Vetrella, Sofia Maria Rosaria Matarese, Bruno Nobili, Sabatino Maione, Silverio Perrotta   +9 more
doaj   +1 more source

Recent Utilization of Pediatric Extrapolation and Modeling and Simulation Approaches in Pediatric Drug Development in Japan

The Journal of Clinical Pharmacology, EarlyView.
Abstract In Japan, the percentage of approved drugs with pediatric indications increased to 30% in 2010‐2015, but no further increase was observed through 2020. The Ministry of Health, Labor, and Welfare in Japan presented draft future directions to promote pediatric drug development, where the modeling and simulation (M&S) approach was introduced as a
Akinori Nakashima, Akihide Tsujimoto, Masako Saito, Jun Takeda, Makiko Natori, Satoshi Shoji   +5 more
wiley   +1 more source

Efficacy and safety of open‐label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study

Journal of Thrombosis and Haemostasis, 2019
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life‐threatening autoimmune thrombotic microangiopathy. Caplacizumab, an anti‐von Willebrand Factor Nanobody®, is effective for treating aTTP episodes and is well tolerated.
P. Knoebl, S. Cataland, F. Peyvandi, P. Coppo, M. Scully, J. K. Kremer Hovinga, A. Metjian, J. de la Rubia, K. Pavenski, Jessica Minkue Mi Edou, H. de Winter, F. Callewaert   +11 more
semanticscholar   +1 more source

Clinical progress note: Rubella

Journal of Hospital Medicine, EarlyView.
Abstract Rates of rubella infection and congenital rubella syndrome decreased significantly since the introduction of the rubella vaccine in 1969. Endemic rubella was declared eliminated in the United States in 2004, and since 2012, all rubella cases in the United States have been associated with infections acquired abroad.
Adam E. Gailani, Walter Dehority, Sophie E. Katz   +2 more
wiley   +1 more source

Evaluation of the diagnostic performance of platelet-derived indices for the differential diagnosis of thrombocytopenia in pediatrics

Revista de la Facultad de Medicina, 2014
Background. Platelet-derived indices have a well-established correlation with the differential diagnosis of thrombocytopenia in adult-based research. These indices include mean platelet volume, platelet distribution width, and platelet-large cell ratio.
Nelson Hernando Aponte Barrios, Adriana Linares Ballesteros, Isabel Cristina Sarmiento Urbina, Gloria Inés Uribe Botero   +3 more
doaj   +1 more source

Graves’ disease-induced immune thrombocytopenic purpura in an African female: a case report

Journal of Medical Case Reports, 2023
Background Immune thrombocytopenic purpura is a condition associated with an unusual, unexplained, and sometimes very severe reduction in the level of platelets in the blood.
Kwabena Oteng Agyapong, Aba A. Folson, Kate Fiador, Cecilia Kootin-Sanwu, Martha Tufuor, Sally Afua Bampoh, Henrietta Fiscian, Roland Wonkyi   +7 more
doaj   +1 more source