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Weak evidence surrounds use of mesenchymal stem cells
A Cochrane review found weak evidence to support limited use of mesenchymal stem cells in hematopoietic transplant.
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High-calorie diet may worsen Wilson disease
The results of the study in rats could explain striking phenotype-genotype discrepancies between patients with Wilson disease, and may give reason...
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Women survive more often than men do when hospitalized with cirrhosis
Women more often had infections and comorbidities, while men more often had liver decompensation, which contributed most significantly to their...
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FDA Expanded Access benefits heavily pretreated patients, especially children
Although approximately 2% of cancer cases at Memorial Sloan Kettering Cancer Center are pediatric, 34.1% of single-patient uses of investigational...
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Gene therapy in hemophilia is just version 1.0
PRAGUE – The biggest question for gene therapy today is durability, according to Dr. John Pasi.
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Think duration, not dose, when managing bleeding with non–factor replacements
PRAGUE – The duration of treatment is more strongly linked to thromboembolism than dose magnitude, according to Dr. Andreas Tiede.
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Emicizumab performs well in surgical setting
PRAGUE – Based on the experience in this small study, bleeding complications were most common during dental procedures.
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Mucinous ovarian tumor survival rates stress correct diagnosis
Women with invasive, well-differentiated mucinous ovarian cancer are more likely to die from their disease within 10 years of diagnosis than women...
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Subcutaneous FVIIa marzeptacog alfa reduced bleeding days
PRAGUE – The treatment was well tolerated with no reports of thrombosis or antidrug antibodies detected.
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Padua variant factor IX gene shines in AMT-061
PRAGUE – Early data shows clinically meaningful factor IX activity in men with hemophilia B.
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SABR response rate falls short in early NSCLC
In a phase 2 trial of patients with resectable stage I NSCLC, stereotactic ablative radiotherapy provided a lower than expected pCR of 60%, and...
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Haplo-HCT shows viability in DLBCL
Survival rates were similar for matched and unmatched donors.
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Adding palbociclib upped responses in previously treated MCL
The positive response means that the combination of ibrutinib and palbociclib will continue to be tested in a phase 2 trial.
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Fitusiran is reversible during dosing suspension
PRAGUE – Temporary suspension of a phase 2 trial of fitusiran offered a chance to study the reversibility of the antithrombin agent.
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Targeted triplet shows potential for B-cell cancers
The nonchemotherapy regimen achieved complete or partial responses in more than 80% of patients in the phase 1 study.