Results 41 to 50 of about 57,561 (211)

Prospective Study of Targeted Busulfan–Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Genetic Rare Diseases

European Journal of Haematology, EarlyView.
ABSTRACT Objectives Genetic rare diseases (GRDs), including chronic granulomatous disease, familial hemophagocytic lymphohistiocytosis, and congenital neutropenia, often require hematopoietic stem cell transplantation (HSCT) as the only curative option.
Bo Kyung Kim, Kyung Taek Hong, Jung Yoon Choi, Hyun Jin Park, Kyung‐Sang Yu, In‐Jin Jang, Hyoung Jin Kang   +6 more
wiley   +1 more source

Thrombotic Microangiopathy‐Like Phenotype in Patients With Infection‐Associated Disseminated Intravascular Coagulation Treated With Thrombomodulin Alfa

European Journal of Haematology, EarlyView.
ABSTRACT Despite disseminated intravascular coagulation (DIC) and thrombotic microangiopathy (TMA) sharing features of thrombocytopenia, organ dysfunction, and bleeding, the relationship between these two conditions remains unclear. We therefore conducted a post hoc analysis of post‐marketing surveillance data from Japan to evaluate the clinical ...
Naoki Takezako, Fumiyo Komatsu, Goichi Honda, Noriaki Kawano, Toshimasa Uchiyama, Kazuo Kawasugi, Seiji Madoiwa, Kei Suzuki, Yoshinobu Seki, Takayuki Ikezoe, Kohji Okamoto, Hideo Wada   +11 more
wiley   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Patient Preferences for the Treatment of Haemophilia A and B in France

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia impacts health and quality of life. Advances in treatments such as factor replacement therapy (FRT), bispecific monoclonal antibody (BS‐mAb), gene therapy (GT), rebalancing treatment and high sustained factor necessitate understanding patient preferences to inform therapeutic strategies. Aim This study quantified trade‐
Yesim Dargaud, Yasmine Fahfouhi, Monia Ezzalfani, Astrid Foix Colonier, Alaeddine Sidhom, Aline Gauthier, Jérémie Rudant, Meriem Kessouri, Nicolas Giraud, Benoît Guillet, Anaïs Reynaud   +10 more
wiley   +1 more source

The XX Factor in Hemophilia: Diagnostic, Therapeutic, and Research Gaps for Women and Girls

Haemophilia, EarlyView.
ABSTRACT Introduction Hemophilia has traditionally been viewed as an X‐linked disorder affecting men and boys, with women and girls labeled as “carriers”, presumed to be clinically unaffected. This paradigm has contributed to under‐recognition, delayed diagnosis, and undertreatment of females with hemophilia‐associated genotypes despite an increasingly
Kelsey Uminski, Ellen Cusano
wiley   +1 more source

Changes in Plasma Levels of Natural Anticoagulants in Disseminated Intravascular Coagulation: High Prognostic Value of Antithrombin and Protein C in Patients with Underlying Sepsis or Severe Infection

Annals of Laboratory Medicine, 2014
Background Dysfunctional natural anticoagulant systems enhance intravascular fibrin for mation in disseminated intravascular coagulation (DIC), and plasma levels of natural anti coagulants can be used in the diagnosis and prognosis of DIC.
Q. Choi, K. Hong, Ji-Eun Kim, H. K. Kim
semanticscholar   +1 more source

From Bleeding Control to Health: Implementing Multidimensional and Multidisciplinary Hemophilia Care Into the Era of Therapeutic Innovation

Haemophilia, EarlyView.
ABSTRACT Introduction Over the past decade, hemophilia management has been transformed by modern prophylactic strategies, achieving dramatic reductions in annualized bleeding rates (ABR), often approaching zero. However, the conceptual evolution of hemophilia care toward multidimensional health assessment and comprehensive multidisciplinary management ...
Nicolas Giraud, Yesim Dargaud
wiley   +1 more source

Complement–Eosinophil Crosstalk Links Complement Activation to Fibrin Deposition in Eosinophilic Chronic Rhinosinusitis

Allergy, EarlyView.
Ryo Hasegawa, Shigeharu Ueki, Misaki Arima, Shohei Nishiyama, Keinosuke Hizuka, Kohei Tatsumi, Ayako Sawai, Anna Shimizu, Yuki Sonoda, Eiichi Kato, Masanori Kidoguchi, Masafumi Sakashita, Yohei Sato, Atsushi Kato, Bruce K. Tan, Robert C. Kern, Robert P. Schleimer, Shigeharu Fujieda, Yoshimasa Imoto   +18 more
wiley   +1 more source

Extended Double Plasma Molecular Adsorption Sessions and Bilirubin Clearance: A Case Report

Hemodialysis International, EarlyView.
ABSTRACT Background The double plasma molecular adsorption system is an artificial‐liver treatment model combining the use of ion exchange resins and neutral microporous resins to remove toxins such as inflammatory cytokines without consuming large amounts of plasma and albumin. Given the relatively expensive consumables needed for Bilirubin adsorption,
Hui Ye, Yi Long Hao, Hai Nan Yang, Wei Fang Yuan, Kai Liang Xu, Xiao Xiao Li, Ming Lei, Yu Xia Sun   +7 more
wiley   +1 more source

Characterization of antithrombin levels in pregnancy.

Thrombosis Research, 2014
OBJECTIVE To characterize antithrombin (AT) levels in normal pregnancy. METHODS We performed secondary analyses with data from 3 studies. Using a single measurement from each subject in the first analysis (cross-sectional), we correlated AT levels with
A. James, Eleanor H. J. Rhee, B. Thames, C. Philipp   +3 more
semanticscholar   +1 more source