Arthritis & Rheumatism, Volume 60,
October 2009 Abstract Supplement
The 2009 ACR/ARHP Annual Scientific Meeting
Philadelphia October 16-21, 2009.
Rapid and Sustained Remission of Macrophage Activation Syndrome (MAS) Associated with Systemic-Onset Juvenile Idiopathic Arthritis (SoJIA) by Treatment with Anakinra but without Cyclosporine
Bruck, Normi, Hamel, Juliane, Suttorp, Meinolf, Gahr, Manfred, Pessler, Frank
To describe the dramatic response of two patients with MAS associated with SoJIA to treatment with Anakinra and a corticosteroid, but without cyclosporine.
We established a protocol for the treatment of MAS associated with SoJIA, allowing for a two week trial of Anakinra and a corticosteroid as initial treatment. Inclusion criteria: SoJIA as defined by the International League of Associations for Rheumatology classification of juvenile idiopathic arthritis (1) and MAS as defined by Ravelli et al. (2).
Between September 2008 and March 2009, two patients met entry criteria.
Patient 1 was an 8-year-old Caucasian boy. At initial diagnosis of SoJIA, treatment with prednisolone and indomethacin led to almost complete resolution of clinical symptoms and normalization of laboratory abnormalities. However, during the prednisolone taper he suddenly developed thrombocytopenia and a dramatic rise in liver transaminases, ferritin, and D-dimers, accompanied by a paradoxically mild elevation of ESR and CRP. A bone marrow aspirate (performed after a 3 day methylprednisolone pulse) showed reduced cellularity and dysfunctional maturation in myelopoiesis, but no hemophagocytosis, but the patient nonetheless satisfied criteria for SoJIA-associated MAS (2). Treatment with oral prednisolone (2 mg/kg/day) and Anakinra (2 mg/kg/d s.c.) led to resolution of fevers and restoration of the patient's sense of well being within 3 days. All lab values normalized within 20 days. At 6 months follow-up, the patient remains in complete remission on monotherapy with Anakinra (2 mg/kg/d s.c.).
Patient 2 was a 12-year-old Caucasian girl. SoJIA-associated symptoms and laboratory abnormalities improved initially under treatment with prednisolone and ibuprofen. At the beginning of the prednisolone taper, the patient developed recurrent fevers, hepatosplenomegaly, general lymphadenopathy, pancytopenia with complete agranulocytosis, and highly elevated ferritin, D-dimer, LDH, ASAT, ALAT, and GGT levels. The bone marrow aspirate showed hemophagocytosis and complete absence of granulopoiesis. Treatment with dexamethasone (10 mg/m2/d) led to a decrease in serum ferritin and liver transaminases, but did not affect fever or agranulocytosis. The patient defervesced within 48 h of starting Anakinra (2 mg/kg/d s.c.), and the granulocyte count normalized within 7 days. All other laboratory indices normalized within 14 days despite a dexamethasone taper. 14 weeks later, complete remission has been sustained on Anakinra monotherapy at a stable dose of 2 mg/kg/d.
Early initiation of IL-1 directed therapy may be effective for the treatment of MAS and could reduce the necessity for potentially toxic long-term treatment with corticosteroids, cyclosporine and/or etoposide.
To cite this abstract, please use the following information:
Bruck, Normi, Hamel, Juliane, Suttorp, Meinolf, Gahr, Manfred, Pessler, Frank; Rapid and Sustained Remission of Macrophage Activation Syndrome (MAS) Associated with Systemic-Onset Juvenile Idiopathic Arthritis (SoJIA) by Treatment with Anakinra but without Cyclosporine [abstract]. Arthritis Rheum 2009;60 Suppl 10 :234