Rationale and efficacy of interleukin-1 targeting in pediatric Erdheim- Chester disease
نویسندگان
چکیده
Result The patient was followed for three years for daily fever 40°C, hepato-splenomegaly, bone pain of the lower limbs and growth retardation. ESR (105mm/h) and Creactive protein level (CRP=97mg/l) were high. Bone X ray found multiple osteolytic and osteosclerotic lesions in femurs, tibia, and pelvis. Whole-body MRI showed hyperintensity of the skeletal bone marrow in T2weighted images and retroperitoneal infiltration. Bone biopsies confirmed ECD. The patient received 3x10 units of IFN-a2a thrice weekly. All symptoms disappeared, ESR and CRP normalized after 4 months. The whole-body MRI showed regression of retroperitoneal infiltration, and improvement of bone marrow intensity. At month 10, the patient experienced a relapse. Serum IL-6 drastically increased 174 pg/ml (N: 0-8.6) but not IL-1 and TNF-a. The patient’s IL-6, IL-1b and TNF-a measurement from supernatant of PBMC culture showed respectively increased levels compared to control, before (71.8ng/ml, 1290pg/ml, 980pg/ml vs 3±1.1, 32±8.9, 174±99) and after stimulation (155.9ng/ml, 3800pg/ml, 1250pg/ml vs 90±11, 3770±506, 1433±196). The patient was then treated with Anakinra, an IL-1 receptor antagonist at 2mg/kg/day. In one week, fever, bone pain resolved, ESR and CRP normalized. The child gained 8 kg weight and 6 cm height in 10-month treatment. MRI at month 7 showed stability of bone lesion and retroperitoneal infiltration. No side effect was notified.
منابع مشابه
Rationale and efficacy of interleukin-1 targeting in Erdheim-Chester disease.
Erdheim-Chester disease (ECD) pathophysiology remains largely unknown. Its treatment is not codified and usually disappointing. Interferon (IFN)-α therapy lacks efficacy for some life-threatening manifestations and has a poor tolerance profile. Because interleukin (IL)-1Ra synthesis is naturally induced after stimulation by IFN-α, we hypothesized that recombinant IL-1Ra (anakinra) might have so...
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