Chediak-Higashi Syndrome Medication
- Author: Roman Janusz Nowicki, MD, PhD; Chief Editor: Dirk M Elston, MD more...
Medication Summary
The goals of pharmacotherapy are to reduce morbidity and to prevent complications.
Antiviral agents
Class Summary
These agents inhibit DNA synthesis and viral replication. Nucleoside analogs are initially phosphorylated by viral thymidine kinase to eventually form a nucleoside triphosphate. These molecules inhibit HSV polymerase with 30-50 times the potency of human alpha-DNA polymerase.
Use as suggested and dosing below is not for antiviral properties but for cellular antimitotic activity at high doses for the accelerated phase.
Acyclovir (Zovirax)
Inhibits activity of both HSV-1 and HSV-2. Has affinity for viral thymidine kinase and once phosphorylated causes DNA chain termination when acted on by DNA polymerase.
Use as suggested and dosing below is not for antiviral properties but for cellular antimitotic activity at high doses for the accelerated phase.
Immune modulators
Class Summary
These agents inhibit key steps in immune reactions.
Immune globulin intravenous (Sandoglobulin, Gammagard, Gamimune, Gammar-P)
Neutralize circulating myelin antibodies through anti-idiotypic antibodies; down-regulates proinflammatory cytokines, including INF-gamma; blocks Fc receptors on macrophages; suppresses inducer T and B cells and augments suppressor T cells; blocks complement cascade; promotes remyelination; may increase CSF IgG (10%).
Interferon alfa-2a and -2b (Roferon-A, Intron A)
Protein product manufactured by recombinant DNA technology. Mechanism of antitumor activity is not clearly understood; however, direct antiproliferative effects against malignant cells and modulation of host immune response may play important roles.
Antineoplastic agents
Class Summary
These agents inhibit cell growth and proliferation. Useful in the accelerated phase of the disease.
Vincristine (Vincasar PFS, Oncovin)
Mechanism of action is uncertain. May involve a decrease in reticuloendothelial cell function or an increase in platelet production. Reduce dose by 50% if direct bilirubin level > 3 mg/100 mL.
Vinblastine (Alkaban-AQ, Velban)
Inhibits microtubule formation, which, in turn, disrupts the formation of the mitotic spindle, causing cell proliferation to arrest at metaphase. Use hematologic parameters as a guide. If direct bilirubin level >3, then reduce dose by 50%.
Local injection of hyaluronidase and application of moderate heat to area of extravasation help disperse drug and are thought to minimize discomfort and possibility of cellulitis.
Anti-inflammatory agents
Class Summary
Systemically interfere with events leading to inflammation.
Colchicine
Decreases leukocyte motility and phagocytosis in inflammatory responses.
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