Results 151 to 160 of about 314,160 (267)

Therapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (alloHSCT) have both demonstrated remissions in VEXAS; however, comparative data is lacking. We conducted a multicenter, retrospective analysis of 66 patients diagnosed with VEXAS syndrome treated with HMA (n = 35) or alloHSCT (n = 31). Baseline characteristics
Saubia Fathima   +48 more
wiley   +1 more source

Quizartinib in Combination With FLAG‐IDA for Relapsed or Refractory Acute Myeloid Leukemia (FLAG‐QUIDA): A PETHEMA Phase I‐II Trial

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Quizartinib is a tyrosine kinase inhibitor with single agent activity in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) and has demonstrated efficacy in first‐line therapy when combined with intensive chemotherapy in both FLT3 ITD‐negative and positive AML.
Teresa Bernal   +29 more
wiley   +1 more source

Sebetralstat for on-demand treatment of hereditary angioedema: A pooled analysis of placebo-controlled clinical trials. [PDF]

open access: yesWorld Allergy Organ J
Aygören-Pürsün E   +40 more
europepmc   +1 more source

Clinical Predictors of Response in Chronic Graft‐Versus‐Host Disease: Results From the “Predicting the Quality of Response to Specific Treatments (PQRST)” Trial

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Despite significant progress in chronic GVHD therapies, challenges remain in understanding pleomorphic phenotypes and varying responses to treatment. The aim of this study was to identify predictors of treatment response. We conducted a prospective, observational cohort study of patients beginning first‐, second‐, or third‐line systemic ...
Betty K. Hamilton   +11 more
wiley   +1 more source

Dordaviprone Maintenance After Allogeneic HCT for High‐Risk Acute Myeloid Leukemia and Myelodysplastic Neoplasm

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Dordaviprone is a first‐in‐class small‐molecule imipridone. In preclinical studies, it is active against leukemia cells harboring a TP53 mutation or complex karyotype and leukemia stem cells while sparing normal bone marrow cells. This study aimed to determine the safety of dordaviprone maintenance after allogeneic hematopoietic cell ...
Vijaya Raj Bhatt   +8 more
wiley   +1 more source

Assessment of noninvasive parameters prior to diagnosis of pulmonary hypertension in patients with fibrotic interstitial lung disease. [PDF]

open access: yesERJ Open Res
Herbert S   +14 more
europepmc   +1 more source

Lymphatic Abnormalities in Noonan Syndrome Extend Beyond Clinically Apparent Disease

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Lymphatic disease represents a well‐described manifestation of Noonan syndrome (NS), yet the full phenotypic spectrum remains incompletely characterized, especially in asymptomatic individuals. We conducted a cross‐sectional study including 10 individuals with NS (four with peripheral lymphedema and six without) and 10 age‐ and sex‐matched ...
Inger Norlyk Sheyanth   +7 more
wiley   +1 more source

Quantifying glare phenomena in patients with different intraocular lenses using a standard and modified clinical straylight meter. [PDF]

open access: yesJ Cataract Refract Surg
Naujokaitis T   +5 more
europepmc   +1 more source

A Novel Splice Variant in ERGIC1 Causes Arthrogryposis Multiplex Congenita—Characterization Using Urine‐Derived Cells

open access: yesAmerican Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Arthrogryposis multiplex congenita (AMC) is defined as the presence of joint contractures affecting at least two body regions at birth. Three different ERGIC1 variants have been reported in individuals with AMC. Here, we report on a 16‐year‐old male with a homozygous ERGIC1 c.250+1G>A variant that was classified as a variant of uncertain ...
Lauren Kerr   +7 more
wiley   +1 more source

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