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.
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.
Department of Nephrology, Kidney Transplantation and Hypertension, The Children's Memorial Health Institute, Warsaw, Poland.
Ryszard
R
Grenda
Department of Nephrology, Kidney Transplantation and Hypertension, The Children's Memorial Health Institute, Warsaw, Poland.
Wioletta
W
Jarmużek
Department of Nephrology, Kidney Transplantation and Hypertension, The Children's Memorial Health Institute, Warsaw, Poland.
Joanna
J
Latoszyńska
Department of Nephrology, Kidney Transplantation and Hypertension, The Children's Memorial Health Institute, Warsaw, Poland.
Sylwester
S
Prokurat
Department of Nephrology, Kidney Transplantation and Hypertension, The Children's Memorial Health Institute, Warsaw, Poland.
Jacek
J
Rubik
D001161:Arteriosclerosis / Q000188:drug therapy* / Q000209:etiology; D001565:Benzoates / Q000627:therapeutic use*; D002648:Child; D004265:DNA Helicases / Q000235:genetics; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use*; D007153:Immunologic Deficiency Syndromes / Q000188:drug therapy* / Q000209:etiology; D016030:Kidney Transplantation / Q000009:adverse effects*; D008297:Male; D009154:Mutation; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000209:etiology; D010009:Osteochondrodysplasias / Q000188:drug therapy* / Q000209:etiology; D010956:Plasmapheresis; D010976:Platelet Count; D000081207:Primary Immunodeficiency Diseases; D011655:Pulmonary Embolism / Q000188:drug therapy* / Q000209:etiology; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000150:complications / Q000188:drug therapy* / Q000601:surgery*; D011720:Pyrazoles / Q000627:therapeutic use*; D013926:Thrombopoietin / Q000378:metabolism; D013997:Time Factors
D001565:Benzoates; D006834:Hydrazines; D011720:Pyrazoles; D013926:Thrombopoietin; C412482:SMARCAL1 protein, human; D004265:DNA Helicases; C520809:eltrombopag
Schimke immuno-osseous dysplasia; acute immune thrombocytopenia; plasmapheresis; renal transplantation; thrombopoietin-receptor agonist