Evaluation of the efficiency of hydroxychloroquine in treating children with immune thrombocytopenia (ITP)

Roche, Ombeline; Aladjidi, Nathalie; Rakotonjanahary, Josué; Leverger, Guy; Leblanc, Thierry; Thomas, Caroline; Pasquet, Marlène; Courcoux, Mary-France; Bayart, Sophie; Gilibert-Yvert, Marion; Neven, Bénédicte; Quartier, Pierre; Pellier, Isabelle

doi:10.1002/ajh.24698

Cited by 9 publications

(15 citation statements)

References 6 publications

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“…Those characteristics guided the choice of hydroxychloroquine, used early and preferentially in 40% of those cases, and allowed satisfying results without any further second-line treatment in 54% of cases. Previous ITP series of small children (n = 28) or adults (n = 27) treated with hydroxychloroquine showed response rates of 50-70% when ANA were positive, without adverse effects (Khellaf et al, 2014;Roche et al, 2017). In this context, our current guidelines now recommend long-term treatment with hydroxychloroquine to prevent the development of SLE, monitored by ophthalmological follow-up and pharmacological dosages.…”

Section: Discussionmentioning

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: Discussionmentioning

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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“…Of note, all included patients received the background therapy with hydroxychloroquine, which has been verified to have a multifaceted effect in primary APS, including the thromboprotective role as well as the potential to lowering aPL levels and increasing platelet count (25)(26)(27). In a most recent pilot open-label randomized prospective study, long-term hydroxychloroquine exposure was related to a decrease in some of aPL titers over an average 2.6-year follow-up (25).…”

Section: Discussionmentioning

confidence: 99%

“…In a most recent pilot open-label randomized prospective study, long-term hydroxychloroquine exposure was related to a decrease in some of aPL titers over an average 2.6-year follow-up (25). For TP therapy, adding hydroxychloroquine to a second-line treatment for TP has been confirmed as an attractive therapeutic approach in the treatment of children with ITP, particularly in the second line after failure of corticosteroids and/ or IVIG (26). In adult patients, the recent study conducted by Khellaf et al (27) similarly revealed that hydroxychloroquine seems to be a safe and efficacious second-line option for patients with SLE-ITP or ITP and high titer of antinuclear antibodies.…”

Section: Discussionmentioning

confidence: 99%

Sirolimus Monotherapy for Thrombocytopenia in Primary Antiphospholipid Syndrome: A Pilot Study From a Tertiary Referral Center

Xie

Zhang

2022

Front. Immunol.

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BackgroundThrombocytopenia (TP) is considered as a warning sign of high-risk antiphospholipid syndrome (APS) and sometimes a paradoxical sign of anti-thrombosis treatment. Currently, there is an extreme paucity of effective and safe drugs for long-term management of TP in primary APS patients; therefore, we explored the efficacy and safety of sirolimus monotherapy.MethodsIn this real-world study, we included 7 consecutive patients with primary APS who received sirolimus monotherapy for TP. Oral sirolimus was initiated at a dose of 1–2 mg once daily and then adjusted primarily based on clinical efficacy and tolerance, with consideration of the sirolimus trough concentration of ≤15 ng/ml.ResultsOf included patients, the median age was 58 years with a median disease course of 1.5 years and 4 patients were treatment-naïve. All patients completed 6 months of sirolimus therapy with a median follow-up of 6 months (range: 6–15). All patients received sirolimus monotherapy for TP during the entire follow-up, without any additional agents. Overall, the platelet count exhibited a substantially increasing trend after sirolimus administration during the first 6 months (p < 0.001) and stability later. Specifically, the median platelet count was significantly increased from 59 × 109/l before sirolimus to 90 × 109/l at month 1 (p = 0.028), 131 × 109/l at 3 months (p = 0.028), and 178 × 109/l at 6 months (p = 0.018). Overall and complete responses were respectively achieved in 6 (85.7%) and 5 (71.4%) patients at month 6. Importantly, overall response was achieved in all 4 treatment-naïve patients. Additionally, there were different extents of decline in the titers of antiphospholipid antibodies after sirolimus treatment. Regarding safety, only one patient experienced an elevated cholesterol level with recovery after atorvastatin treatment.ConclusionSirolimus monotherapy confers good efficacy and tolerance for TP in primary APS patients and therefore may be considered as a first-line therapy.

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“…Two French retrospective studies, one in adults ( n = 28) and one in children ( n = 46) suggested the effectiveness of hydroxychloroquine patients with primary ITP and ANA positivity with a response (platelet count ≥30 × 10 9 /L, doubling from baseline values and no bleeding) rate of 50.0% in adults and 56.5% in children. However, there was no comparator (ANA‐negative patients) in these studies 24,25 …”

Section: Association Of Antinuclear Antibodies and Itp Phenotypementioning

confidence: 99%

“…However, there was no comparator (ANA-negative patients) in these studies. 24,25 In a retrospective multicentric study in China, the presence of ANAs at a titre ≥1:80 was detected in 98 (34.1%) of 287 adult patients treated with rituximab at a median duration of ITP of 6 months (range: 3-460). The rate of overall response (platelet count ≥30 × 10 9 /L, doubling from baseline values and no bleeding) was 76.5% in the ANApositive group and 55.0% in the ANA-negative group.…”

Section: Response To Itp Treatmentsmentioning

confidence: 99%

Clinical significance of antinuclear antibodies in primary immune thrombocytopenia

Moulis,

Aladjidi,

Godeau

2023

Br J Haematol

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SummaryThere are discrepancies across guidelines about whether the dosage of antinuclear antibodies (ANAs) is of use at the diagnosis of primary immune thrombocytopenia (ITP). This review describes the current knowledge about ANA prevalence in patients with primary ITP, and their potential usefulness as biomarkers for ITP evolution, response to treatments and increased risk of subsequent development of systemic lupus and thrombosis.

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Evaluation of the efficiency of hydroxychloroquine in treating children with immune thrombocytopenia (ITP)

Cited by 9 publications

References 6 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

Sirolimus Monotherapy for Thrombocytopenia in Primary Antiphospholipid Syndrome: A Pilot Study From a Tertiary Referral Center

Clinical significance of antinuclear antibodies in primary immune thrombocytopenia

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