Results 281 to 290 of about 180,819 (342)

Bleeding Disorders in Children With Genetic Diseases: A Narrative Review

Acta Paediatrica, EarlyView.
ABSTRACT Aim The lack of data on bleeding risk assessment in children with genetic diseases is concerning given their increased care needs and risk of haemorrhagic complications compared to the general population. Identification of haemostatic disorders is crucial for implementing preventive measures and mitigating bleeding risk.
Raphaelle Cagol, Mariam Sbeity Barakat, Marjolaine Willems, Tasnime Akbaraly, Biron‐Andreani Christine, Isabelle Diaz, Alexandre Theron   +6 more
wiley   +1 more source

Fatal viscerotropic and neurotropic disease after yellow fever vaccine: a rare manifestation leading to diagnosis of severe combined immunodeficiency in an infant. [PDF]

Rev Inst Med Trop Sao Paulo
Vieira LJT, Goebel GA, Barcelos Y, Cunha LO, Santos LTM, Romanelli RMC, Minafra FG, Carvalho AL, Carvalho LFA, Diniz LMO.   +9 more
europepmc   +1 more source

Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. [PDF]

Clin Immunol
Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Dávila Saldaña BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E.   +56 more
europepmc   +1 more source

Phase II study of the triple combination of rabbit ATG, ciclosporin and eltrombopag in patients with transfusion‐dependent aplastic anaemia: West Japan Hematology Study Group (W‐JHS) AA02 trial

British Journal of Haematology, EarlyView.
Summary The efficacy of a triple combination of rabbit anti‐human thymocyte immunoglobulin (rATG), ciclosporin and eltrombopag (EPAG) was prospectively evaluated in patients with severe or transfusion‐dependent non‐severe aplastic anaemia (SAA) across 29 institutions in Japan. Sixty patients were enrolled, of whom 48 had SAA. The primary end‐point, the
Fumi Nakamura, Ken Ishiyama, Ritsuro Suzuki, Hiroyuki Maruyama, Yasushi Onishi, Kensuke Usuki, Hideo Koh, Katsuto Takenaka, Ryunosuke Saiki, Seishi Ogawa, Kana Matsumoto, Takuto Takahashi, Kazuyoshi Hosomichi, Yoshitaka Zaimoku, Kenichi Yoshimura, Itaru Matsumura, Koichi Akashi, Shinji Nakao   +17 more
wiley   +1 more source

Severe combined immunodeficiency.

Indian pediatrics, 2009
openaire   +1 more source

Severe Combined Immunodeficiency from a Homozygous DNA Ligase 1 Mutant with Reduced Catalytic Activity but Increased Ligation Fidelity. [PDF]

J Clin Immunol
Alajlan H, Raducanu VS, Lopez de Los Santos Y, Tehseen M, Alruwaili H, Al-Mazrou A, Mohammad R, Al-Alwan M, De Biasio A, Merzaban JS, Al-Mousa H, Hamdan SM, Alazami AM.   +12 more
europepmc   +1 more source

Preclinical <i>ex vivo</i> IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease. [PDF]

Genes Dis
Hu M, Xu Q, Zhang F, Buckland KF, Gao Y, Du W, Ding Y, Zhou L, Sun X, Ma L, Zhang Z, Tang X, Zhao X, Thrasher AJ, An Y.   +14 more
europepmc   +1 more source

X-linked severe combined immunodeficiency complicated by disseminated bacillus Calmette-Guérin disease caused by a novel pathogenic mutation in exon 3 of the IL2RG gene: a case report and literature review. [PDF]

Front Immunol
Jiang C, He Y, Chen X, Xia F, Shi F, Xu X, Sun T, You K.   +7 more
europepmc   +1 more source

Position statement from the British Society of Blood and Marrow Transplantation and Cellular Therapy on insertional oncogenesis in gene‐engineered advanced cell therapy products for treatment of haematological disorders

British Journal of Haematology, EarlyView.
S. Ghorashian, R. Sanderson, J. Lee, A. Black, M. A. O'Reilly, D. I. Marks, J. Snowden, D. Richardson, E. Olavarria   +8 more
wiley   +1 more source

Safety and effectiveness of the combination of 5‐azacitidine and ruxolitinib in VEXAS syndrome: A single‐centre experience

British Journal of Haematology, EarlyView.
Gregorio Maria Bergonzi, Enrico Cozzo, Alessandro Tomelleri, Costanza Piccolo, Gianluca Scorpio, Carmelo Gurnari, Francesca Romano, Marco Matucci‐Cerinic, Lorenzo Dagna, Massimo Bernardi, Luca Vago, Fabio Ciceri, Corrado Campochiaro, Elisa Diral   +13 more
wiley   +1 more source