Medication Summary
Glycogen-storage disease type Ia (GSD Ia) has no specific medication requirement beyond prophylactic PO iron and prompt treatment of intercurrent infections (which may interrupt PO intake).
Weekly administration of granulocyte colony–stimulating factor (GCSF), in addition to prophylactic PO iron and prompt treatment of intercurrent infections, is critical in patients with glycogen-storage disease type Ib. GCSF administration is now standard therapy to prevent or reduce incidences of serious infection. GCSF may also delay or prevent pseudocolitis symptoms. A 2014 multicenter study indicated that bone mineralization status correlated positively with initial age and duration of GCSF administration. [14]
Trace elements
Class Summary
These are inorganic substances found in small amounts in the tissues and required for various metabolic processes.
Iron sulfate (Feosol)
Nutritionally essential inorganic substance.
Colony stimulating factors
Class Summary
These agents act as hematopoietic growth factors that stimulate the development of granulocytes. They are used to treat or prevent neutropenia when patients are receiving myelosuppressive cancer chemotherapy and to reduce neutropenia associated with bone marrow transplantation. These drugs are also used to mobilize autologous peripheral blood progenitor cells for bone marrow transplantation and to manage chronic neutropenia.
Filgrastim (G-CSF, Neupogen)
GCSF that activates and stimulates production, maturation, migration, and cytotoxicity of neutrophils.
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Microsome is shown in relation to the substrate, glucose-6-phosphate, which has been released from cytosolic glycogen. This substrate is transferred across the microsomal membrane by the protein translocase, where by glucose-6-phosphatase acts on it to release free glucose and inorganic phosphate. Patients with glycogen-storage disease type Ia are genetically deficient in glucose-6-phosphate activity, while those affected with glycogen-storage disease type Ib lack translocase.
