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Central point: THE use of mavorixafor (400 mg, oral, 1/day) in WHIM patients increased the total number of white blood cells, neutral granulocytes and lymphocyte counts;
summary: Warts, hypoproglobulinemia, infection and myelin anemia syndrome (WHIM) are rare primary immunodeficiencys caused by mutations in the function of the CXCR4 gene.
Mavorixafor is a select small molecule antagonist for oral CXCR4 subjects that increases the mobilization and transport of white blood cells from bone marrow sources.
(Functional Acquisition Mutation of CXCR4 Receptor) Dale and others conducted an open-label, incremental, extended Phase 2 trial to assess the safety, toerability, pharmacodynamics, pharmacodynamics, and initial activity of mavorixafor for WHIM syndrome.
recruited a total of 8 patients with WHIM syndrome who had been genetically tested and treated with mavorixafor in doses increasing to 400 mg (1/day).
5 patients entered the extended queue for 28.6 months of continuous treatment.
(the chemical structure of Mavorixafor) Mavorixafor is well-resistant and has no treatment associated with severe adverse reactions.
16.5 months of medium follow-up, the researchers observed an increase in dose dependence in absolute neutral granulocyte count (ANC) and absolute lymphocyte count (ALC).
the dose reaches 300 mg/day, the ANC can be maintained at more than 500 /μL, with a medium of 12.6 hours, while the ALC can be maintained at more than 1000 mg/L for up to 16.9 hours.
follow-up to the extended queue found that the annual infection rate in patients at effective doses dropped to 2.27 from 4.63 in the 12 months before the trial.
, the number of skin warts observed decreased by an average of 75%.
(significant reduction in skin warts after 18 months of Mavorixafor treatment), the trial showed that mavorixafor (400 mg, 1/day) mobilized neutral granulocytes and lymphocytes in adult patients with WHIM syndrome, and long-term treatment provided initial clinical efficacy in such patients.
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