Management strategies for newly diagnosed immune thrombocytopenia in Italian AIEOP Centres: do we overtreat? Data from a multicentre, prospective cohort study.


Journal

Blood transfusion = Trasfusione del sangue
ISSN: 2385-2070
Titre abrégé: Blood Transfus
Pays: Italy
ID NLM: 101237479

Informations de publication

Date de publication:
09 2020
Historique:
received: 13 02 2020
accepted: 21 05 2020
entrez: 15 9 2020
pubmed: 16 9 2020
medline: 7 5 2021
Statut: ppublish

Résumé

The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated. During a period of 1 year, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, history, clinical symptoms, platelet count and treatment at presentation and at subsequent visits. Of the 205 patients, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years; p<0.001) and had lower platelet counts. At the 3-month follow-up, 92/202 patients (46%) had persistent ITP. Recovery within 3 months was predicted by younger median age (5.3 vs 7.8 years; p<0.001), and recent viral infection (p<0.001) . At 1 year, 56 patients had chronic ITP, which was associated with older median age (7.54 vs 5.35 years; p<0.001), and a family history of autoimmunity (p<0.05; relative risk: 1.81; 95% confidence interval: 1.09-2.98). In total, 357 pharmacological treatments were recorded (216 intravenous immunoglobulins, 80 steroids). Response to intravenous immunoglobulins did not have an effect on remission rate at 12 months. Pediatric hematologists in Italian Centre treat over three-quarters of patients with newly diagnosed ITP, despite recent international guidelines. Almost 80% of patients with mild clinical symptoms received pharmacological treatment at diagnosis, which was significantly associated with younger age. Chronicity at 12 months was not affected by different therapeutic approaches at diagnosis or response to therapy.

Sections du résumé

BACKGROUND
The aim of the present study was to assess management strategies for immune thrombocytopenia (ITP) among Italian paediatric haematologists, and to compare these with those of recent international guidelines. Predictors of early remission or disease chronicity were also evaluated.
MATERIALS AND METHODS
During a period of 1 year, 205 children (age: 1 month-18 years) with newly diagnosed ITP were prospectively enrolled by 16 centres belonging to the Italian Association of Paediatric Haematology and Oncology (AIEOP). We collected the subjects demographic data, history, clinical symptoms, platelet count and treatment at presentation and at subsequent visits.
RESULTS
Of the 205 patients, 47 (23%) were initially managed with a wait-and-see approach. Compared to these patients, children administered platelet-enhancing therapies were significantly younger (median age: 4.75 vs 7.96 years; p<0.001) and had lower platelet counts. At the 3-month follow-up, 92/202 patients (46%) had persistent ITP. Recovery within 3 months was predicted by younger median age (5.3 vs 7.8 years; p<0.001), and recent viral infection (p<0.001) . At 1 year, 56 patients had chronic ITP, which was associated with older median age (7.54 vs 5.35 years; p<0.001), and a family history of autoimmunity (p<0.05; relative risk: 1.81; 95% confidence interval: 1.09-2.98). In total, 357 pharmacological treatments were recorded (216 intravenous immunoglobulins, 80 steroids). Response to intravenous immunoglobulins did not have an effect on remission rate at 12 months.
DISCUSSION
Pediatric hematologists in Italian Centre treat over three-quarters of patients with newly diagnosed ITP, despite recent international guidelines. Almost 80% of patients with mild clinical symptoms received pharmacological treatment at diagnosis, which was significantly associated with younger age. Chronicity at 12 months was not affected by different therapeutic approaches at diagnosis or response to therapy.

Identifiants

pubmed: 32931416
pii: 2020.0041-20
doi: 10.2450/2020.0041-20
pmc: PMC7592159
doi:

Substances chimiques

Immunoglobulins, Intravenous 0

Types de publication

Clinical Trial Comparative Study Journal Article Multicenter Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

396-405

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Auteurs

Emilia Parodi (E)

Haematology Unit, Department of Paediatric and Public Health Sciences, University of Turin, Turin, Italy.

Giovanna Russo (G)

Paediatric Haematology and Oncology Unit, "Policlinico-Vittorio Emanuele" Hospital, University of Catania, Catania, Italy.

Piero Farruggia (P)

Paediatric Haematology and Oncology Unit, A.R.N.A.S. Civic Hospital, Palermo, Italy.

Lucia D Notarangelo (LD)

Onco-Haematology and Bone Marrow Transplantation Unit, Children's Hospital, Brescia, Italy.

Maria T Giraudo (MT)

Department of Mathematics, University of Turin, Turin, Italy.

Margherita Nardi (M)

Paediatric Haematology Oncology, Bone Marrow Transplant, "S. Chiara" University Hospital, Pisa, Italy.

Fiorina Giona (F)

Department of Translational and Precision Medicine, "Sapienza" University, Rome, Italy.

Paola Giordano (P)

Department of Biomedical Sciences and Human Oncology, Pediatric Section, "A. Moro" University of Bari, Bari, Italy.

Ugo Ramenghi (U)

Haematology Unit, Department of Paediatric and Public Health Sciences, University of Turin, Turin, Italy.

Angelica Barone (A)

Department of Paediatric Onco-Haematology, University Hospital, Parma, Italy.

Gianluca Boscarol (G)

Department of Paediatrics, Central Teaching Hospital Bolzano, Bolzano, Italy.

Simone Cesaro (S)

Paediatric Haematology Oncology, University Hospital, Verona, Italy.

Francesca Fioredda (F)

Clinical and Experimental Unit, "G. Gaslini" Children's Hospital, Genoa, Italy.

Saverio Ladogana (S)

Department of Haematology, "IRCCS Casa Sollievo della Sofferenza", San Giovanni Rotondo, Italy.

Maria Licciardello (M)

Paediatric Haematology and Oncology Unit, "Vittorio Emanuele" Polyclinic, University of Catania, Italy.

Francesca Rossi (F)

"Santobono Pausilipon" Hospital, Naples, Italy.

Laura Rubert (L)

Paediatric Haematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.

Marco Spinelli (M)

MBBM Foundation, Department of Paediatrics, University of Milano-Bicocca, Monza, Italy.

Fabio Tucci (F)

Department of PaediatricOnco-Haematology, Meyer Children's Hospital, Florence, Italy.

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