scholarly journals A phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixafor

Blood ◽  
2014 ◽  
Vol 123 (15) ◽  
pp. 2308-2316 ◽  
Author(s):  
David H. McDermott ◽  
Qian Liu ◽  
Daniel Velez ◽  
Lizbeeth Lopez ◽  
Sandra Anaya-O’Brien ◽  
...  

Key Points Plerixafor can be given safely to WHIM syndrome patients twice daily for a 6-month period and appears promising as a treatment.

Blood ◽  
2016 ◽  
Vol 127 (4) ◽  
pp. 411-419 ◽  
Author(s):  
Harriet S. Walter ◽  
Simon A. Rule ◽  
Martin J. S. Dyer ◽  
Lionel Karlin ◽  
Ceri Jones ◽  
...  

Key Points We report a first-in-human dose-escalation study in relapsed/refractory B-cell malignancies with the potent BTK inhibitor ONO/GS-4059. ONO/GS-4059 induced clinically durable responses in relapsed/refractory B-cell malignancies without significant toxicities.


Blood ◽  
2018 ◽  
Vol 131 (25) ◽  
pp. 2836-2845 ◽  
Author(s):  
Myrna R. Nahas ◽  
Robert J. Soiffer ◽  
Haesook T. Kim ◽  
Edwin P. Alyea ◽  
Jon Arnason ◽  
...  

Key Points Costimulatory blockade using abatacept represents a novel therapeutic approach for the treatment of cGVHD. Abatacept resulted in a clinical response in 44% of patients with both decreased prednisone use and T-cell PD-1 expression in responders.


2013 ◽  
Vol 18 (5) ◽  
pp. 20-26
Author(s):  
E. G Deeva ◽  
Leg Ivanovich Kiselev ◽  
Tatjana Ilinichna Melnikova ◽  
Aram Arutjunovich Shaldzhan ◽  
Petr Alexeevich Nekrasov ◽  
...  

In the work there are presented results of the study of the pharmacokinetics, tolerability and safety of a new anti-influenza preparation Triazavirin in several dose regimes, performed in 30 volunteers. There were no recorded adverse reactions associated with the administration of the drug. Study of the pharmacokinetics showed that the maximum concentration of Triazavirin in blood plasma is achieved on average 1 - 1.5 hours after, the curve of decreasing concentrations is two-phase, half-life elimination period in the first phase is 0.5 - 1.4 hour. It is impossible to exclude polymorphic character of metabolism. The extent of systemic exposure depends on the dosage frequency more than the dose of the drug. In this case, there was no noted significant accumulation in long-term use. In the study Triazavirin was well tolerated by all participants in all the studied dose regimes. The preferred regime, providing the maintenance of an effective and safe concentrations is recognized as dosage of the 250 mg twice a day.


Author(s):  
J.E. Meyer ◽  
T.J. Galloway ◽  
K. Ruth ◽  
E.A. Ross ◽  
M.K. Buyyounouski ◽  
...  

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