Second‐line treatments in children with immune thrombocytopenia: Effect on platelet count and patient‐centered outcomes

Grace, Rachael F.; Shimano, Kristin A.; Bhat, Rukhmi; Neunert, Cindy; Bussel, James B.; Klaassen, Robert J.; Lambert, Michele P.; Rothman, Jennifer A.; Breakey, Vicky R.; Hege, Kerry; Bennett, Carolyn M.; Rose, Melissa J.; Haley, Kristina M.; Buchanan, George R.; Geddis, Amy E.; Lorenzana, Adonis; Jeng, Michael; Pastore, Yves D.; Crary, Shelley E.; Neier, Michelle; Neufeld, Ellis J.; Neu, Nolan; Forbes, Peter; Despotovic, Jenny M.

doi:10.1002/ajh.25479

Cited by 35 publications

(40 citation statements)

References 16 publications

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“…For 55% of children, second‐line treatment with curative intent was administered, in some patients up to 13 years after diagnosis. Contrary to a recently published experience, a majority of patients from our cohort (65%) did not receive a second‐line treatment prior to being diagnosed with cITP (Grace et al , 2018, 2019). There is no evidence to guide the sequence of treatment.…”

Section: Discussioncontrasting

confidence: 99%

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

Gourdonneau

Fernandes³

et al. 2020

Br J Haematol

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Summary Childhood chronic immune thrombocytopenic purpura (cITP) is a rare disease. In severe cases, there is no evidence for the optimal therapeutic strategy. Our aim was to describe the real‐life management of non‐selected children with cITP at diagnosis. Since 2004, patients less than 18 years old with cITP have been enrolled in the national prospective cohort, OBS’CEREVANCE. From 1990 to 2014, in 29 centres, 392 children were diagnosed with cITP. With a median follow‐up of six years (2·0–25), 45% did not need second‐line therapy, and 55% (n = 217) received one or more second lines, mainly splenectomy (n = 108), hydroxychloroquine (n = 61), rituximab (n = 61) or azathioprine (n = 40). The overall five‐year further second‐line treatment‐free survival was 56% [95% CI 49·5–64.1]. The use of splenectomy significantly decreased over time. Hydroxychloroquine was administered to children with positive antinuclear antibodies, more frequently older and girls, and reached 55% efficacy. None of the patients died. Ten years after the initial diagnosis, 55% of the 56 followed children had achieved complete remission. Children with cITP do not need second‐line treatments in 45% of cases. Basing the treatment decision on the pathophysiological pathways is challenging, as illustrated by ITP patients with positive antinuclear antibodies treated with hydroxychloroquine.

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Section: Discussioncontrasting

confidence: 99%

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

Gourdonneau

Fernandes³

et al. 2020

Br J Haematol

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“…Studies of children have suggested that fatigue may also be a feature of childhood ITP (Blatt et al , ; Sarpatwari et al , ; Grace et al , ) and have described a negative impact of ITP on HRQoL (reviewed by Trotter & Hill, ; Grace et al , ). Anecdotally, some parents also report mood changes or behavioural problems in their children following a diagnosis of ITP.…”

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confidence: 99%

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

et al. 2020

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Childhood ITP is often considered to be a relatively mild haematological disorder, with only a minority of patients requiring treatment for troublesome bleeding. Over recent years, wider effects of the condition have been identified in some adults, particularly relating to fatigue and cognitive impairment. In this study, we sought to investigate such effects in a group of children with ITP and further our understanding of their psychological profile. Children attending routine haematology outpatient clinics and their parents were asked to complete standardised questionnaires designed to assess a range of psychological and cognitive factors. Although the majority of children had some scores within the normal range, a significantly high proportion had difficulties with fatigue (70Á6%), emotional and behavioural symptoms (25Á7%) or executive functioning (19Á4%). Quality of life and subjective evaluation of the illness (appraisal) correlated significantly with each of these domains, but bleeding severity and platelet count did not. Our findings provide valuable insight into the broader impact of childhood ITP, which could aid in providing holistic care, potentially contribute to decisions regarding medical treatment, and guide future research.

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“…Moreover, 52% of patients used rituximab or other immunosuppressant drugs. However, this study involved a significant proportion of children with newly diagnosed or secondary ITP (16).…”

Section: Discussionmentioning

confidence: 99%

“…Corticosteroids and intravenous immunoglobulins (IVIG) are recommended as first-line treatments. If first-line therapy fails, therapeutic options for managing chronic ITP include immunosuppressive drugs (such as rituximab, mycophenolate mofetil, and sirolimus) (2,(16)(17)(18)(19)(20)(21), splenectomy or, more recently, thrombopoietin receptor agonists (TPO-RAs) (22,23).…”

Section: Introductionmentioning

confidence: 99%

Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

et al. 2020

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Second‐line treatments in children with immune thrombocytopenia: Effect on platelet count and patient‐centered outcomes

Abstract: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder with isolated thrombocytopenia and hemorrhagic risk. While many children with ITP can be safely observed, treatments are often needed for various reasons, including to decrease

Cited by 35 publications

References 16 publications

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

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

Fatigue, executive function and psychological effects in children with immune thrombocytopenia: a cross‐sectional study

Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP)

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