Managing Childhood Immune Thrombocytopenia in a Government Facility in India; Perspective from an LMIC

Abstract

Background and Aims: The pathophysiology and treatment of immune cytopenia, especially Immune thrombocytopenia (ITP) has evolved in recent times. Childhood ITP is mostly self-limiting, where spontaneous recovery is the norm. In this study, we evaluated the treatment and follow up data of children with ITP treated at our centre Methods: Clinical, haematological and outcome data of patients aged 0-18 years with ITP between April 2017- March 2022 (60 months) were analysed. Results: 144 patients were registered. The median age was 7 years (0.3-18 years). M:F was 1.52:1. Initial platelet count ranged from <1000 to 62,000/mL. Bone marrow evaluation was done in 92 patients. 2 were excluded for an alternative diagnosis (TTP, CAMT). 1 patient's data was not available. Treatment was given to those with platelet count <20,000/mL or mucosal bleeding. Standard definitions were followed. Out of 142 patients with ITP, 82 (57%) were acute/newly diagnosed, 14 had persistent, 37 (26%) had chronic disease and 9 had recurrent presentations with intermittent remissions. All patients received Prednisolone as first agent. 51 patients (from persistent and chronic) received Dapsone. 3rd line included other immunosuppressives, rituximab and TPO agonists. The median follow-up was for 40 months (3-54 months). 67% are in remission and 3 were lost to follow-up after remission. 20 attained partial response (platelet count >10,000/mL) and are either on no treatment or continued on dapsone. 6 patients were LTFU before remission was achieved. 7 patients have no response, continue to have platelet count <10,000/mL. 3 children have WAS, 1 XLT, 1 Biotinidase deficiency and 1 combined immunodeficiency. 1 child each has psoriasis, leukoderma, HIV and 2 have lupus. Conclusions: The natural history of the disease still remains to be one of spontaneous remissions. Among chronic and persistent ITP, alternative diagnoses emerged. Dapsone response in >60% chronic cases.