Second Line ITP Therapy in the UK: Results from the UK Paediatric ITP Registry
Notice bibliographique
Résumé
Abstract Background: Immune thrombocytopaenia (ITP) is the most common acquired bleeding disorder in children and is typically a self-limiting condition that resolves within six months. Although a 'watch and wait' approach is commonly used in the management of paediatric ITP, treatment options are available to aid in managing complex or severe cases, with the ultimate goal being to maintain health-related quality of life (HRQL) and avoid serious complications. This study aims to understand the use and indications for second-line treatments in persistent and chronic ITP (duration of 3 months or longer) based on an analysis of the UK Paediatric ITP Registry. Methods: The UK Paediatric ITP Registry is a prospective database of children presenting with ITP to over 100 paediatric treatment centres in the UK between 2005 and 2015. Patients with ITP were included if they received any second line ITP treatment: specifically rituximab, thrombopoietin receptor agonists (TPO-RAs), azathioprine, dapsone and splenectomy. Factors including age, sex, bleeding episodes and severity as well as follow-up platelet counts were analysed. Patients receiving second line treatments were then compared to similar patients who did not receive treatment but who also had persistent (> 3 months) or chronic (> 12 months) ITP and at least one platelet count of 30x109/l or less during that time period. Results: Of 938 patients in the database, 537 were identified as having persistent or chronic ITP. 22 of these patients received a form of second line treatment. This is 4% of all persistent and chronic ITP patients and 16% of those with at least one recorded platelet count <30x109/l. Of the patients receiving second line treatment, 77% were female and 59% were age 10 or older when diagnosed, which when compared to the control group, show age (p=0.002) and sex (p=0.019) as significant factors in the use of second line treatment. Bleeding episodes and severity were similar between both groups with 64% of patients in each group having at least one episode of moderate or severe bleeding. Of the 22 patients receiving second line treatment, 59% showed a marked improvement in platelet counts after one course. 32% showed no significant response despite multiple courses (Figure). Conclusion: In the UK Paediatric ITP Registry, teenage girls were more likely to be prescribed second line therapy (50% were females aged 10 or older), and the majority of patients showed a marked improvement in their platelet count with one course of therapy. Future research should look at the impact of TPO-RAs as they become licensed and HRQL outcomes to help select which ITP patients would benefit the most from second line therapy. Figure 1. Graphic representation of platelet counts throughout course of ITP in patients receiving second line treatments using a logarithmic scale Figure 1. Graphic representation of platelet counts throughout course of ITP in patients receiving second line treatments using a logarithmic scale Disclosures No relevant conflicts of interest to declare.
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Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,012 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,001 |
| Bibliométrie | 0,003 | 0,008 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,001 | 0,001 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».