Eltrombopag (thrombopoietin‐receptor agonist) and plasmapheresis as rescue therapy of acute post‐renal transplant immune thrombocytopenia in a child with Schimke immuno‐osseous dysplasia—case report

Abstract

SIOD is rare disorder related to SMARCAL1 or SMARCAL2 gene mutation, including (among other comorbidities) T-cell immunodeficiency, nephrotic syndrome, and renal failure. Up to 22% of primary patients may develop various autoimmune disorders. We report the case of 11-year-old male with SIOD, who presented ITP at 2 years after renal transplantation with decrease in platelet count (from normal) to 56 000/μL and then (gradually) to 2000/μL. There was no effect of iv. methylprednisolone/dexamethasone. As the presence of antibodies against GPIIb/IIIa, GPIb, and GPIaIIa platelet glycoproteins was confirmed, patient was given 50 g of IVIG and then was put on plasmapheresis; however, both showed poor direct effect. As we were afraid to give rituximab (due to expected overimmunosuppression), we prescribed the oral TPO-receptor agonist (eltrombopag). Patient responded after 17 days of therapy, to the final dose of 50 mg/d (approx. 2 mg/kg). The antiplatelet antibodies disappeared after four plasmapheresis. Overall, the therapy was continued for 7 weeks and was stopped at platelet count of 433 000/μL. Platelet count remained stable in 8-month follow-up. Combination of plasmapheresis and TPO-receptor agonist was effective in post-renal transplant acute ITP in patient with SIOD.

Drug-Induced Hematological Cytopenia in Kidney Transplantation and the Challenges It Poses for Kidney Transplant Physicians

“…They failed to improve platelet count [ 293 ]. Yet in another case report eltrombopag and plasmapheresis were successfully used as rescue therapy of acute post-renal transplant immune thrombocytopenia in a child with Schimke immuno-osseous dysplasia [ 294 ]. Further evidence is needed for its usefulness and safety in drug-induced thrombocytopenia in context of kidney transplantation.…”

Schimke immuno-osseous dysplasia in a boy with generalized edema; a case report

“…Schimke immuno-osseous dysplasia is a rare multisystemic autosomal recessive disease. The SIOD accounts for about one in 1 000 000 to 3 000 000 live births in the US (8)(9)(10)(11). The etiology of SIOD needs to be clarified in more detail.…”

“…The SIOD accounts for about one in 1 000 000 to 3 000 000 live births in the US (8)(9)(10)(11). Standard clinical and laboratory manifestations in SIOD include steroidresistant nephrotic syndrome (SRNS), progressive renal failure, T-cell immunodeficiency, spondyloepiphyseal dysplasia, and hypothyroidism.…”

Schimke immuno-osseous dysplasia: A case report

“…Transient ischemic attacks, cerebral infarction, atherosclerosis, and migraine-like headache are common neurologic manifestations of SIOD. [234] In this article, we describe a 4.5-year-old boy who presented with nephrotic syndrome and other extra renal manifestations from Iran.…”