Horse ATG(ATGAM) versus rabbit ATG (Fresenius) for treatment of aplastic anaemia in children: results of prospective double-blind randomised single-centre trial

M.A. Maschan, G. Novichkova, D.D. Baidildina, E.V. Suntzova, E.G. Kravchenko, O.V. Goronkova, L.I. Zharikova, M.M. Schneider, N.Y. Bogatcheva, A.A. Maschan

Author address: 

Russian Children's Hospital (Moscow, RUS)


Background: Combined IST with horse ATG and CsA has become a standard therapy for young patients with severe aplastic anemia (SAA) lacking HLA-compatible family donor. Rabbit ATG(Thymoglobuline)  has been used in Europe for second-line therapy in non-responders and relapsing patients, but randomized comparison of horse vs. rabbit ATG has not been performed. Objective: To compare in a randomized fashion efficacy of horse ATG (ATGAM, Upjohn) vs rabbit ATG (ATG-Fresenius, Fresenius) for pediatric SAA and VSAA patients. Patients and methods: Thirty-two patients with untreated SAA and VSAA, mean age 10.8y (2.5-18y), M/F ratio 25:7, were randomized to receive either ATGAM 160 mg/kg (15 pts) or ATG-Fresenius 40 mg/kg (17 pts). Methylprednisolone 1 mg/kg was given for 14 days starting the day  of 1-st ATG infusion. Patient accrual was started in December 2000 and finished in March 2003. Pts with vSAA comprised 7 (46%) in the ATGAM group and 11(65%) in the ATG-Fresenius group. Mean ANC count was 344 /mm3  and 348/ mm3  in ATGAM and Fresenius group respectively.  M:F ratio was 9:6 for ATGAM group and 16:1 for Fresenius group. Mean age was 11.9y in the ATGAM and 9.8 in the Fresenius group. All patients received cyclosporine A (Neoral) at starting dose 5 mg/kg/day, with trough level targeted to 100-300ng/ml. rhG-CSF was used only in pts with active infection uncontrolled by standard antimicrobial therapy. Primary end-point was probability of hematologic response at 6 mo, defined as transfusion independence with Hb > 90 g/l, plt > 20x109/l and ANC > 0.5x109/l with at least doubling from the baseline. Relapse rate within one year of hematologic response, overall and event-free survival will be evaluated at follow-up. Patients not responding to first ATG course were crossed-over to the second study drug at six months. Results. Both ATGs were equally well tolerated. One of the recipients of ATGAM died of aspergillosis within one mo after treatment and 14 pts were evaluable, as were all 17 pts in ATGFresenius group. In the ATGAM group 8 of 8 SAA pts responded as did 5 of 6 vSAA pts (RR-93%).  In the ATG Fresenius 3 of 6 SAA pts and 5 of 11 vSAA achieved HR (RR-47%). p

abstract No: 


Full conference title: 

30th Annual Meeting of the European Group for Blood and Marrow Transplantation
    • EBMT 2004