Second‐line treatment trends and long‐term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years

Ducassou, Stéphane; Gourdonneau, Anne; Fernandes, Helder; Leverger, Guy; Pasquet, Marlène; Fouyssac, Fanny; Bayart, Sophie; Bertrand, Yves; Gautier, Michel; Jeziorski, Éric; Thomas, Caroline; Abouchallah, Wadih; Viard, F; Guitton, Corinne; Cheikh, Nathalie; Pellier, Isabelle; Carausu, Liana; Droz, Cécile; Leblanc, Thierry; Aladjidi, Nathalie

doi:10.1111/bjh.16448

Cited by 18 publications

(29 citation statements)

References 54 publications

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“…The higher rate of sustained CR in ITP among patients with pES compared to patients with cITP alone may be due to more patients with pES receiving treatment. 18 …”

Section: Discussionmentioning

confidence: 99%

Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden

Pincez

Fernandes²,

Leblanc

et al. 2021

haematol

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Pediatric-onset Evans syndrome (pES) is defined by both immune thrombocytopenic purpura (ITP) and autoimmune hemolytic anemia (AIHA) before the age of 18 years. There have been no comprehensive long-term studies of this rare disease, which can be associated to various immunopathological manifestations (IMs). We report outcomes of the 151 patients with pES and more than 5 years of follow-up from the nationwide French prospective OBS’CEREVANCE cohort. Median age at final follow-up was 18.5 (6.8–50.0) years and the median follow-up period was 11.3 (5.1–38.0) years. At 10 years, ITP and AIHA were in sustained complete remission in 54.5% and 78.4% of patients, respectively. The frequency and number of clinical and biological IMs increased with age: at 20 years old, 74% had at least one clinical cIM. A wide range of cIMs occurred, mainly lymphoproliferation, dermatological, gastrointestinal/hepatic and pneumological IMs. The number of cIMs was associated with a subsequent increase in the number of second-line treatments received (other than steroids and immunoglobulins; hazard ratio, 1.4; 95% confidence interval, 1.15–1.60; p = 0.0002, Cox proportional hazards method). Survival at 15 years after diagnosis was 84%. Death occurred at a median age of 18 (1.7–31.5) years, and the most frequent cause was infection. The number of second-line treatments and severe/recurrent infections were independently associated with mortality. In conclusion, longterm outcomes of pES showed remission of cytopenias but frequent IMs linked to high secondline treatment burden. Mortality was associated to drugs and/or underlying immunodeficiencies, and adolescents-young adults are a high-risk subgroup.

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“…The higher rate of sustained CR in ITP among patients with pES compared to patients with cITP alone may be due to more patients with pES receiving treatment. 18 …”

Section: Discussionmentioning

confidence: 99%

Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden

Pincez

Fernandes²,

Leblanc

et al. 2021

haematol

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“…It could reverse T cell abnormalities in patients who have responded to therapy. [ 13 ] Previous studies showed that clinical symptoms in patients with chronic immune thrombocytopenia might not relieve by using EPAG alone. [ 14 ] Rituximab was used in a number of clinical treatments, including hematological malignancies, autoimmune diseases, and organ transplantation.…”

Section: Discussionmentioning

confidence: 99%

Early treatment of rituximab combined with eltrombopag for secondary thrombocytopenic purpura in rheumatoid arthritis

Zhang

Bao

et al. 2022

Medicine

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Rationale: Secondary immune thrombocytopenic purpura (ITP) is also known as acquired thrombocytopenic purpura, autoimmune disease is usually one of the important causes. There are few reports about treatment of refractory thrombocytopenic purpura in rheumatoid arthritis (RA). We report a case of refractory ITP in which changes in platelet-related markers with therapeutic agents are worthy of the attention of clinicians. Patient concerns: A 69-year-old woman admitted for ecchymosis on the neck and arms for 15 days presented to our hospital. She was diagnosed with RA 5 years ago. Diagnosis: The diagnosis met the American College of Rheumatology/European League Against Rheumatism 2010 classification criteria. The disease activity score 28 (DAS-28) was 4.6, indicating that the disease activity was moderate. Interventions: Treatment with first-line therapies and second-line treatment--eltrombopag (EPAG) were ineffective. Therefore, we performed rituximab combined with a low dose of EPAG. Outcomes: The patient received 2 cycles of rituximab combined with EPAG, and reported no new petechiae on her buccal mucosa and limbs during follow-up. Lessons: This case suggests that early treatment of rituximab combined with EPAG is beneficial to patients with refractory ITP in RA. In terms of disease dynamic monitoring, immature platelet fraction (IPF) may be an auxiliary indicator for predicting efficacy, but its significance needs further study.

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“…Since 2004, French patients with cITP diagnosed before 18 years of age have been included in the prospective OBS'CEREVANCE cohort. 9,17 The inclusion and exclusion criteria for the cohort are presented in Table S1. Written informed consent was obtained from all participants.…”

Section: Obs'cerevance Cohortmentioning

confidence: 99%

“…6 Pediatric cITP is associated with poor health-related quality of life, 7,8 and second-line treatments (i.e., other than corticosteroids and intravenous immunoglobulins) are required in up to 55% of patients. 9 Disease remission is common and almost 75% of cITP patients with pediatric-onset seem to undergo spontaneous remission during 5 years of follow-up. 6,10,11 Some patients with cITP have associated immunopathological manifestations (IMs), clinical IMs (such as lymphoproliferative disorder and organ autoimmunity), and/or biological IMs (such as antinuclear antibodies [ANA] and hypogammaglobulinemia).…”

Section: Introductionmentioning

confidence: 99%

“…6,10,11 Some patients with cITP have associated immunopathological manifestations (IMs), clinical IMs (such as lymphoproliferative disorder and organ autoimmunity), and/or biological IMs (such as antinuclear antibodies [ANA] and hypogammaglobulinemia). 9 However, the prevalence and spectrum of IMs in pediatric cITP are unknown. As in Evans syndrome, 12,13 the occurrence of IMs suggests an underlying condition, classically systemic lupus erythematosus (SLE) or primary immunodeficiency (PID).…”

Section: Introductionmentioning

confidence: 99%

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Impact of age at diagnosis, sex, and immunopathological manifestations in 886 patients with pediatric chronic immune thrombocytopenia

Pincez

Fernandes

Pasquet³

et al. 2023

American J Hematol

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Pediatric chronic immune thrombocytopenia (cITP) is a heterogeneous condition in terms of bleeding severity, second‐line treatment use, association with clinical and/or biological immunopathological manifestations (IMs), and progression to systemic lupus erythematosus (SLE). No risk factors for these outcomes are known. Specifically, whether age at ITP diagnosis, sex, or IMs impact cITP outcomes is unknown. We report the outcomes of patients with pediatric cITP from the French nationwide prospective cohort OBS'CEREVANCE. We used multivariate analyses to investigate the effect of age at ITP diagnosis, sex, and IMs on cITP outcomes. We included 886 patients with a median (min‐max) follow‐up duration of 5.3 (1.0–29.3) years. We identified an age cutoff that dichotomized the risk of the outcomes and defined two risk groups: patients with ITP diagnosed <10 years (children) and ≥ 10 years (adolescents). Adolescents had a two to four‐fold higher risk of grade ≥3 bleeding, second‐line treatment use, clinical and biological IMs, and SLE diagnosis. Moreover, female sex and biological IMs were independently associated with higher risks of biological IMs and SLE diagnosis, second‐line treatment use, and SLE diagnosis, respectively. The combination of these three risk factors defined outcome‐specific risk groups. Finally, we showed that patients clustered in mild and severe phenotypes, more frequent in children and adolescents, respectively. In conclusion, we identified that age at ITP diagnosis, sex, and biological IMs impacted the long‐term outcomes of pediatric cITP. We defined risk groups for each outcome, which will help clinical management and further studies.

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Second‐line treatment trends and long‐term outcomes of 392 children with chronic immune thrombocytopenic purpura: the French experience over the past 25 years

Cited by 18 publications

References 54 publications

Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden

Long term follow-up of pediatric-onset Evans syndrome: broad immunopathological manifestations and high treatment burden

Early treatment of rituximab combined with eltrombopag for secondary thrombocytopenic purpura in rheumatoid arthritis

Impact of age at diagnosis, sex, and immunopathological manifestations in 886 patients with pediatric chronic immune thrombocytopenia

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