Results 181 to 190 of about 140,652 (287)
ABSTRACT In clinical trials, it is critical to incorporate prognostic variables to improve precision for estimating treatment effects. Per FDA guidance, these prognostic variables need to be prospectively specified in the statistical analysis plan. Therefore, there is a great need to effectively identify prognostic variables from historical studies ...
Xueying Liu +3 more
wiley +1 more source
Genotypic distribution and molecular spectrum of rare and novel thalassemia variants in Ganzhou, southern China. [PDF]
Huang J, Huang X, Xie X, Xin X.
europepmc +1 more source
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi +3 more
wiley +1 more source
Nanoplate Digital PCR for Identification of α<sup>0</sup>-Thalassemia (SEA Deletion): Carrier Screening and Possible Application to Prenatal Diagnosis. [PDF]
Pattrakorn A +7 more
europepmc +1 more source
Summary Cerebral macrovasculopathy (CV) is a major complication in children with sickle cell anaemia (SCA) and usually requires a long‐term transfusion programme (TP) to prevent stroke. This study aimed to identify factors predicting reversal of CV on TP in a single‐centre newborn cohort. Among 375 patients, 50 presented CV and received TP.
Julie Sommet +16 more
wiley +1 more source
Development and Internal Validation of a Machine Learning-Based Model for Thalassemia Identification Using Complete Blood Count Parameters. [PDF]
Yin P, Tan J, Hong H.
europepmc +1 more source
Summary In patients with sickle cell disease (SCD), parvovirus B19 infection (B19V) leads to acute anaemia (aplastic crisis), but may also be associated with other serious complications. We retrospectively analysed clinical data from paediatric SCD patients with B19V infections between 2023 and 2025, including symptoms, laboratory parameters ...
Matthias Bleeke +42 more
wiley +1 more source
Hemoglobinopathies and Thalassemia in Northeast India: A Scoping Review of Disease Burden, Mutation Spectrum, and Gaps in Molecular Genetic Evidence. [PDF]
Pathak MS +4 more
europepmc +1 more source
ABSTRACT Endocrine mucin‐producing sweat gland carcinoma (EMPSGC) is an adnexal neoplasm which typically occurs on periorbital skin and demonstrates overlapping histopathologic features with primary mucinous carcinoma of the skin (MCS). Herein, we report a patient who developed five distinct lesions of EMPSGC and MCS over an eight‐year period, some of ...
Ikuko Hirai +5 more
wiley +1 more source

