Pegfilgrastmin in Children with Severe Aplastic Anemia during Immunosuppressive Therapy.

Severe neutropenia and infections connected with leukopenia are same of the main complications in immunosuppressive therapy (IST) of severe aplastic anemia (SAA). Granulocyte-colony-stimulating factors are currently given as supportive therapy in SAA protocol treatment beside antithymocyte globulin and cyclosporine A (CSA). We present four cases of girls aged 6–16 years with SAA without family bone marrow donors, who received pegfilgrastmin in IST. The protocol included: antithymocyte globulin (rabbit) 3.75mg/kg/bw iv for 5 days, CSA 5mg/kg/bw orally for 180 days and pegfilgrastmin (Neulasta-Amgen) 6mg in patients over 45kg and 100μg/kg in children less than 45 kg every 10 days sc until absolute neutrophil count (ANC)reached > 1.5x10⁹/l. Blood was monitored every 7 days during therapy. Two patients received 4 doses of pegfilgrastmin. After this therapy the ANC level was normal without any decreases and we observed slow, gradual rise ANC in this girls In these patients there was a complete remission on day 180 of treatment and both are still in remission month 34. The other two patients received 6 doses of pegfilgrastmin. We observed fairly rapid rise and fall of leukocytes during the first 30 days of therapy. The ANC level became normal on day 70 of treatment. One of the patients is now in partial remission day 320. The other girl did not respond to the first course of IST and she received the second course of IST due to the lack of an unrelated bone marrow donor. Pegfilgrastmin was also administered during this therapy. After 3 doses of pegfilgrastmin leukocyte level rose to 5.3x10⁹/l and ANC level to 2.3x10⁹/l. At present the patient is in partial remission and is still receiving CSA day 210. Pegfilgrastimn administered every 10 days was well tolerated by all four patients and there were no adverse events. We observed that pegfilgrastmin was as effective as filgrastmin which was previously administered. No changes were noted during ANC recovery. However administration of pegfilgrastmin reduced the number of injections given and improved the patient’s well-being.