By Applications : Chronic Inflammatory Demyelinating, Hypogammaglobulinemia
Multifocal Motor Neuropathy, Immunodeficiency Diseases, CVID (common variable immunodeficiency), SCI (severe combined immunodeficiency), Primary Humoral Immunodeficiency, Unknown (idiopathic) Immunodeficiency diseases, Myasthenia Gravis, Guillain-Barre syndrome, MFS (Miller Fisher Syndrome), AMAN (Acute motor axonal neuropathy) and AMSAN (acute motor-sensory axonal neuropathy), Kawasaki disease, and ITP among others.
- The European Commission, based on a favourable recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products, has granted orphan drug designation to mavorixafor (X4P-001) for the treatment of WHIM (Warts, Hypogammaglobulinemia
, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene, US-based biopharmaceutical company X4 Pharmaceuticals, Inc.
Transfer of passive immunity can be evaluated by measuring the serum concentration of total protein; levels below 5 d/dL are associated with hypogammaglobulinemia
X4 Pharmaceuticals announced the initiation of 4WHIM, a pivotal Phase 3 global clinical trial of mavorixafor, X4P-001, for the treatment of Warts, Hypogammaglobulinemia
, Infections, and Myelokathexis, or WHIM, syndrome, a rare, inherited, primary immunodeficiency disease.
: B-cell aplasia and hypogammaglobulinemia
Variations in QoL and the degree of psychological difficulties were found between specific diagnostic groups, with children affected by transient hypogammaglobulinemia
of infancy being amongst those with the lowest scores of QoL Stephenson et al.
LOW-DOSE TREATMENT with plerixafor, a CXC chemokine receptor 4 antagonist, was well tolerated and markedly improved severe presentations of warts, hypogammaglobulinemia
, infections, and myelokathexis (WHIM) syndrome in three patients who could not receive granulocyte colony-stimulating factor (G-CSF) therapy.
Suggestive findings are hypoproteinemia, hypoalbuminemia, hypogammaglobulinemia
The first report on LRBA deficiency was performed in five patients from four consanguineous families with hypogammaglobulinemia
, autoimmune manifestations and recurrent infections (2).
(16) As in adults, thymomas many present with paraneoplastic syndromes including myasthenia gravis, hypogammaglobulinemia
or red cell aplasia.
Poorly responsive Standard therapy does not 8 to standard imply anticancer drugs, but therapy includes corticoids Hypogammaglobulinemia
, monoclonal or oligoclonal paraproteinemia, or absolute lymphocyte count do not by themselves constitute an indication for therapy.