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Compostella, Alessia (2013) Analisi di nuove strategie terapeutiche per pazienti in età pediatrica affetti da tumori solidi refrattari alla chemioterapia standard. [Tesi di dottorato]

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Abstract (inglese)

The PhD research work was performed at the Pediatric Hemato-Oncology Department of Padua University, one of the major AIEOP (Associazione Italiana di Emato-Oncologia Pediatrica) Centres and the coordinating centre of the Soft Tissue Sarcoma Committee (STSC) protocol EpSSG 2005 for pediatric soft tissue sarcomas. The primary goal of the PhD was analyzing new therapeutic strategies for resistant solid tumors children. The work moved from data analysis on adolescents with rhabdomyosarcoma (RMS) and resistant RMS patients treated with a new second line topotecan (T)/carboplatin (C) based regimen, through a specific training in clinical trials management to obtain the necessary expertise to write down a phase II trial for resistant RMS patients. In the last decades in many types of cancer the survival rates are reported to be less favorable in adolescents compared with younger children. To investigate whether this is true for adolescents with RMS, the results obtained in patients enrolled in protocols run by the Italian STSC were analyzed. Our study concluded that the higher prevalence of unfavorable tumor characteristics among adolescents seems to explain their worse outcome, the limited number of adolescents enrolled in STSC studied is worrisome and cooperation with adult oncologists needs to be improved. Another bad result is about prognosis of children with metastatic or resistant RMS: there is a strong need to find new strategies to improve the outcome of these patients. T and C are known to have activity against a variety of pediatric tumors so a T/C based chemotherapy has been proposed as second line CT for children relapsed after being treated in the STSC protocols. Our study shows that the T/C combination is tolerable in heavily pretreated patients; the response rate (RR) is somewhat lower when compared to other combinations tested in phase II studies but it’s of interest for the population with alveolar subtype. Clinical trials are one of the most important tools to explore new therapeutic approaches; for this reason the PhD involved a specific training in clinical trials management through participation to courses ad hoc, creation of a team committed to new drugs environment, participation to international phase II and III trials. During the last part of PhD the efforts were coordinated to write a phase II trial on treatment of leptomeningeal dissemination by RMS. Neoplastic meningitis is a devasting complication of both solid and hematologic tumors and despite treatment the median survival duration is in the range of 8-16 weeks. Among available therapeutic approaches intrathecal (IT) chemotherapy is one of the most widely used even if no impact on survival has been demonstrated. Few anticancer agents are used in this setting, therefore it’s essential to develop new IT agents with novel mechanism of action. Topotecan showed interesting results, then we designed a phase II study with IT topotecan in patients with RMS and EPNET tumors and leptomeningeal spread.

Abstract (italiano)

L’attività di ricerca del dottorato si è svolta press il Dipartimento di Pediatria dell’Università di Padova; l’ unità di Oncoematologia pediatrica è uno dei maggiori centri AIEOP (Associazione Italiana di Emato-Oncologia Pediatrica) ed è il centro coordinatore per il protocollo EpSSG 2005 del comitato sarcomi tessuti molli per la cura dei sarcomi. L’obiettivo del dottorato era analizzare nuove strategie terapeutiche in bambini con tumori solidi recidivi o resistenti. Il lavoro si è svolto passando per l’analisi di dati su una popolazione di pazienti adolescenti affetti da rabdomiosarcoma (RMS), l’analisi di dati su pazienti con RMS resistente trattati con un regime basato su topotecan/carboplatino, una formazione specifica sui trials clinici per giungere all’obiettivo della stesura di un protocollo di fase II su RMS resistenti. Negli ultimi anni è emerso come in molte neoplasie gli adolescenti vadano peggio rispetto ai bambini; per capire se ciò è vero anche per i pazienti adolescenti con RMS, sono stati analizzati i risultati ottenuti dai pazienti con RMS trattati con i protocolli del STSC. Lo studio concludeva che l’outcome peggiore è giustificato per gli adolescenti da una maggior incidenza di caratteristiche prognostiche sfavorevoli, che il tasso di arruolamento nei protocolli è insoddisfacente e pertanto urge una migliore collaborazione con gli oncologi dell’adulto. La prognosi dei pazienti con RMS metastatico o resistente resta negativa a tutt’oggi: cè una forte esigenza di identificare nuove strategie per migliorare la sopravvivenza di questi pazienti. Topotecan e Carboplatino sono farmaci con nota efficacia in vari tumori pediatrici pertanto la loro combinazione è stata proposta come terapia di seconda linea nei bambini che ricadono dopo trattamento secondo i protocolli del STSC. Il nostro studio ha dimostrato che la combinazione è ben tollerata, che i tassi di risposta sono leggermente inferiori rispetto ad altri regimi precedentemente studiati ma interessanti in particolare per l’istotipo alveolare. I trials clinici sono uno degli strumenti più validi per esplorare nuove strategie terapeutiche; per questo buona parte dell’attività di dottorato è stata dedicata a una formazione specifica nella gestione dei trials clinici, attraverso la partecipazione a corsi, la formazione di un gruppo dedicato ai nuovi farmaci, la partecipazione a trials nazionali e internazionali di fase II e III. Durante l’ultima parte del Dottorato gli sforzi sono stati coordinati alla stesura di un protocollo di fase II per il trattamento delle meningosi da RMS/PNET. La meningite neoplastica è una complicanza devastante di neoplasie sia solide che ematologiche; indipendentemente dal trattamento la sopravvivenza si aggira sulle 8-16 settimane. Tra i vari trattamenti la chemioterapia intratecale (CT IT) è molto usata nonostante non vi sia dimostrazione che impatti sulla sopravvivenza. Vi sono pochi chemioterapici ad uso IT disponibili, quindi è necessario trovarne altri. Topotecan sembra promettente in tal senso, pertanto abbiamo disegnato un protocollo di fase II sull’uso di topotecan IT in bambini e adolescenti affetti da RMS/PNET con disseminazione leptomeningea.

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Tipo di EPrint:Tesi di dottorato
Relatore:compostella, a
Correlatore:Bisogno, Gianni
Dottorato (corsi e scuole):Ciclo 24 > Scuole 24 > MEDICINA DELLO SVILUPPO E SCIENZE DELLA PROGRAMMAZIONE > EMATOONCOLOGIA, IMMUNOLOGIA E GENETICA
Data di deposito della tesi:27 Giugno 2013
Anno di Pubblicazione:27 Giugno 2013
Parole chiave (italiano / inglese):rhabdomyosarcoma; rabdomiosarcoma; neoplastic meningitis; meningite neoplastica; topotecan; intrathecal chemotherapy; terapia intratecale.
Settori scientifico-disciplinari MIUR:Area 06 - Scienze mediche > MED/38 Pediatria generale e specialistica
Struttura di riferimento:Dipartimenti > Dipartimento di Medicina
Centri > Centro per lo studio e la cura della leucemia e malattie affini
Codice ID:6127
Depositato il:07 Nov 2014 14:29
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