Response to rituximab in children and adults with immune thrombocytopenia (ITP)

Abstract Background Rituximab is a monoclonal anti‐CD20 antibody used as a second‐line treatment for immune thrombocytopenia (ITP). As additional treatments for ITP emerge, identifying the most appropriate patients and optimal timing for rituximab are important but challenging without established pr...

Full description

Bibliographic Details
Main Authors: Emily M. Harris, Kirsty Hillier, Hanny Al‐Samkari, Laura Berbert, Rachael F. Grace
Format: Article
Language:English
Published: Elsevier 2021-08-01
Series:Research and Practice in Thrombosis and Haemostasis
Subjects:
Online Access:https://doi.org/10.1002/rth2.12587
_version_ 1827840996240523264
author Emily M. Harris
Kirsty Hillier
Hanny Al‐Samkari
Laura Berbert
Rachael F. Grace
author_facet Emily M. Harris
Kirsty Hillier
Hanny Al‐Samkari
Laura Berbert
Rachael F. Grace
author_sort Emily M. Harris
collection DOAJ
description Abstract Background Rituximab is a monoclonal anti‐CD20 antibody used as a second‐line treatment for immune thrombocytopenia (ITP). As additional treatments for ITP emerge, identifying the most appropriate patients and optimal timing for rituximab are important but challenging without established predictors of response to therapy. Objectives The purpose of this study was to describe demographic, clinical, and laboratory characteristics of pediatric and adult patients with ITP to identify differences in evaluation before rituximab administration and correlates of platelet response. Methods This is a retrospective cohort study describing the characteristics of patients with ITP treated with rituximab from 2010 to 2020 at two academic tertiary care centers. Results A total of 64 patients met criteria for inclusion. Complete rituximab response (56%) was not significantly different between children (58%, n = 24) and adults (55%, n = 40). Response rate was similar in those with primary versus secondary ITP (53% vs 62%). Among patients treated with rituximab, Evans Syndrome was more common in children than adults (42% vs 18%). Immunologic labs assessed before rituximab varied by age and were more commonly evaluated in children (lymphocyte subsets 88% vs 22%). Immunologic markers, including antinuclear antibody, direct antiglobulin testing, immunoglobulin levels, and lymphocyte subsets, did not predict response to rituximab in pediatric or adult patients with ITP. Conclusions Pre‐rituximab immunologic evaluation varied significantly between adults and children, which could represent institution‐specific practice patterns or a more general practice difference. If the latter, underlying immunodeficiency in adults with ITP may be underrecognized. Standardized guidance for pre‐rituximab immunologic evaluation is needed.
first_indexed 2024-03-12T07:45:10Z
format Article
id doaj.art-a2e9bef51c5143aa89d32ce199d6be0f
institution Directory Open Access Journal
issn 2475-0379
language English
last_indexed 2024-03-12T07:45:10Z
publishDate 2021-08-01
publisher Elsevier
record_format Article
series Research and Practice in Thrombosis and Haemostasis
spelling doaj.art-a2e9bef51c5143aa89d32ce199d6be0f2023-09-02T21:03:38ZengElsevierResearch and Practice in Thrombosis and Haemostasis2475-03792021-08-0156n/an/a10.1002/rth2.12587Response to rituximab in children and adults with immune thrombocytopenia (ITP)Emily M. Harris0Kirsty Hillier1Hanny Al‐Samkari2Laura Berbert3Rachael F. Grace4Department of Pediatrics Boston Children's HospitalBoston Combined Residency Program Boston MA USAHarvard Medical School Boston MA USAHarvard Medical School Boston MA USAClinical Research Center Boston Children's Hospital Boston MA USAHarvard Medical School Boston MA USAAbstract Background Rituximab is a monoclonal anti‐CD20 antibody used as a second‐line treatment for immune thrombocytopenia (ITP). As additional treatments for ITP emerge, identifying the most appropriate patients and optimal timing for rituximab are important but challenging without established predictors of response to therapy. Objectives The purpose of this study was to describe demographic, clinical, and laboratory characteristics of pediatric and adult patients with ITP to identify differences in evaluation before rituximab administration and correlates of platelet response. Methods This is a retrospective cohort study describing the characteristics of patients with ITP treated with rituximab from 2010 to 2020 at two academic tertiary care centers. Results A total of 64 patients met criteria for inclusion. Complete rituximab response (56%) was not significantly different between children (58%, n = 24) and adults (55%, n = 40). Response rate was similar in those with primary versus secondary ITP (53% vs 62%). Among patients treated with rituximab, Evans Syndrome was more common in children than adults (42% vs 18%). Immunologic labs assessed before rituximab varied by age and were more commonly evaluated in children (lymphocyte subsets 88% vs 22%). Immunologic markers, including antinuclear antibody, direct antiglobulin testing, immunoglobulin levels, and lymphocyte subsets, did not predict response to rituximab in pediatric or adult patients with ITP. Conclusions Pre‐rituximab immunologic evaluation varied significantly between adults and children, which could represent institution‐specific practice patterns or a more general practice difference. If the latter, underlying immunodeficiency in adults with ITP may be underrecognized. Standardized guidance for pre‐rituximab immunologic evaluation is needed.https://doi.org/10.1002/rth2.12587childrenimmune thrombocytopeniaITPrituximabtreatment
spellingShingle Emily M. Harris
Kirsty Hillier
Hanny Al‐Samkari
Laura Berbert
Rachael F. Grace
Response to rituximab in children and adults with immune thrombocytopenia (ITP)
Research and Practice in Thrombosis and Haemostasis
children
immune thrombocytopenia
ITP
rituximab
treatment
title Response to rituximab in children and adults with immune thrombocytopenia (ITP)
title_full Response to rituximab in children and adults with immune thrombocytopenia (ITP)
title_fullStr Response to rituximab in children and adults with immune thrombocytopenia (ITP)
title_full_unstemmed Response to rituximab in children and adults with immune thrombocytopenia (ITP)
title_short Response to rituximab in children and adults with immune thrombocytopenia (ITP)
title_sort response to rituximab in children and adults with immune thrombocytopenia itp
topic children
immune thrombocytopenia
ITP
rituximab
treatment
url https://doi.org/10.1002/rth2.12587
work_keys_str_mv AT emilymharris responsetorituximabinchildrenandadultswithimmunethrombocytopeniaitp
AT kirstyhillier responsetorituximabinchildrenandadultswithimmunethrombocytopeniaitp
AT hannyalsamkari responsetorituximabinchildrenandadultswithimmunethrombocytopeniaitp
AT lauraberbert responsetorituximabinchildrenandadultswithimmunethrombocytopeniaitp
AT rachaelfgrace responsetorituximabinchildrenandadultswithimmunethrombocytopeniaitp