Il portale delle malattie rare e dei farmaci orfani

Therapeutic indication

*Replacement therapy in adults, and children and adolescents (0-18 years) in:
- Primary immunodeficiency syndromes with impaired antibody production,
- Hypogammaglobulinaemia and recurrent bacterial infections in patients with chronic lymphocytic leukaemia, in whom prophylactic antibiotics have failed,
- Hypogammaglobulinaemia and recurrent bacterial infections in plateau phase multiple myeloma patients who have failed to respond to pneumococcal immunisation,
- Hypogammaglobulinaemia in patients after allogeneic haematopoietic stem cell transplantation (HSCT).

*Immunomodulation in adults, and children and adolescents (0-18 years) in:
- Primary immune thrombocytopenia (ITP), in patients at high risk of bleeding or prior to surgery to correct the platelet count,
- Guillain Barré syndrome,
- Kawasaki disease,
- Multifocal Motor Neuropathy (MMN).