Oral Prednisolone Produces a Durable Response in Pediatric Myelodysplastic Syndromes (MDS).

Treatment options for children with MDS are limited except for allogeneic bone marrow transplantation. We present our experience with the use of long duration corticosteroids in children with MDS. Fifty five children (age< 15 years) with pediatric MDS or myelofibrosis treated between 1991–2004 with corticosteroids were analyzed. All of these patients presented with cytopenia involving one or more lineages and a hypercellular marrow with dysplasia and increased reticulin. Prednisolone was started at 1 mg/kg daily or on alternate days for a period of 1–2 months. If there was no response, steroids were rapidly tapered and stopped. In children who showed a response, the steroids were slowly tapered to alternate day steroids and stopped over a period of 6–12 months. In patients who had relapse of symptoms on tapering, the dose of steroids was increased to previous levels and then tapered at a much slower rate. The median age was 3 years (range: 3 months – 14 years) with 36 males and 19 females. Forty six has pediatric MDS while 10 had pediatric myelofibrosis. The median duration of symptoms prior to starting steroids was 7 months (range: 1–24). Forty one patients (74.5%) showed a response with 31 (56.4%) showing a complete response while 10 patients (18.1%) showed a partial response with improvement in counts and transfusion independence. Fourteen patients (25.5%) showed no improvement and continued to be transfusion dependant. Fourteen of the 41 patients (34%) who responded to steroids relapsed on tapering of steroids but showed response to repeat courses of steroids. The median duration of steroid use in patients who have responded is 12 months (range: 1 – 96). None of the patients had any serious side effects including infections, hypertension or diabetes except one patient who developed disseminated tuberculosis 12 months after stopping steroids. Nine patients have shown progression of disease while 1 patient developed features of SLE 24 months after diagnosis of MDS. At a median follow up of 34 months (range: 4– 120), 32 patients (58%) are alive with 27 (49%) in complete remission [14 off treatment, 13 on treatment] and 5 in partial remission but transfusion independent. Prednisolone induces a durable response in children with MDS. The biological basis of this response needs to be evaluated.