Multicenter randomized, double-blind controlled trial to evaluate the efficacy of laser therapy for the treatment of severe oral mucositis induced by chemotherapy in children: laMPO RCT

Objectives: To demonstrate the efficacy of laser photobiomodulation (PBM) compared to that of placebo on severe oral mucositis (OM) in pediatric oncology patients. The primary objective was the reduction of OM grade (World Health Organization [WHO] scale) 7 days after starting PBM. Secondary objectives were reduction of pain, analgesic consumption, and incidence of side effects. Methods: One hundred and one children with WHO grade\ua0>\ua02 chemotherapy-induced OM were enrolled in eight Italian hospitals. Patients were randomized to either PBM or sham treatment for four consecutive days (days +1 to +4). On days +4, +7, and +11, OM grade, pain (following a 0\u201310 numeric pain rating scale, NRS) and need for analgesics were evaluated by an operator blinded to treatment. Results: Fifty-one patients were allocated to the PBM group, and 50 were allocated to the sham group. In total, 93.7% of PBM patients and 72% of sham patients had OM grade\ua0<\ua03 WHO on day +7 (P\ua0=\ua00.01). A significant reduction of pain was registered on day +7 in the PBM versus sham group (NRS 1 [0\u20133] vs. 2.5 [1\u20135], P\ua0<\ua00.006). Reduced use of analgesics was reported in the PBM group, although it was not statistically significant. No significant adverse events attributable to treatment were recorded. Conclusions: PBM is a safe, feasible, and effective treatment for children affected by chemotherapy-induced OM, as it accelerates mucosal recovery and reduces pain

[Computerized tomography of the skull in children with lymphoblastic leukemia treated with prophylactic cranial radiation].

Thirty asymptomatic patients with acute lymphoblastic leukemia who had received prophylactic cranial irradiation (16 pts had 2400 cGy, 14 pts 1800 cGy) and intrathecal methotrexate were studied by computed tomography of the brain 60 to 148 months after initiation of prophylaxis. Three of 30 (10%) patients presented abnormal findings: widening of frontal subarachnoid space (1 patient), little area of decreased attenuation coefficient (1 patient), and intracerebral calcifications (1 patient Tomography abnormalities could be detected either in patients treated with 2400 cGy and in those treated with 1800 cGy. None of our patients showed central nervous system dysfunctions on physical examination. The results of our study suggest that tomography findings have a poor clinical significance

Long term observation of children with idiopathic thrombocytopenic purpura.

N/

VALUTAZIONE QUANTITATIVA DI MALATTIA MINIMA NEL MIDOLLO E NEL SANGUE PERIFERICO DI PAZIENTI CON RABDOMIOSARCOMA MEDIANTE TECNICA DI REAL TIME PCR

Introduzione e scopo. Il Rabdomiosarcoma (RMS), tumore a piccole cellule rotonde blu, \ue8 il pi\uf9 comune sarcoma dei tessuti molli pediatrici. In circa 20% dei casi si presenta con malattia diffusa alla diagnosi e le sedi pi\uf9 frequentemente coinvolte sono polmoni, linfonodi e midollo osseo (BM). Attualmente, l\u2019infiltrazione midollare di cellule di RMS \ue8 rilevata dall\u2019 analisi morfologica dell\u2019 aspirato midollare e/o delle biopsie osteo-midollari. Questi metodi tuttavia non riescono a rilevare un\u2019infiltrazione neoplastica inferiore al 5%. Sulla base delle caratteristiche cliniche e biologiche del RMS, abbiamo standardizzato un metodo quantitativo di RT-PCR basato sul marcatore RMS-specifico MyoD1, al fine di studiare la malattia minima disseminata(MMD) in bambini con RMS. Materiali e metodi. Abbiamo condotto studi di RT-PCR quantitativa relativa per il gene MyoD1 mediante tecnologia TaqMan. I primers specifici per MyoD1 e la sonda sono stati individuati usando il software specifico Primer Express (Applied Biosystems). GUS \ue8 stato utilizzato come gene housekeeping ossia come controllo endogeno per ogni campione. Risultati. Abbiamo studiato il BM alla diagnosi di 39 pazienti affetti da RMS. La metodica \ue8 di facile applicazione ed unisce l\u2019alta sensibilit\ue0 alla specificit\ue0. MMD era positiva in 17/39 pazienti. I livelli di espressione di MyoD1 in 7/17 campioni, positivi anche mediante RT-PCR qualitativa, hanno mostrato valori di Ct che variavano in un range da 21 a 27, mentre in 10 casi, negativi in RT-PCR, il valore di Ct era superiore a 35. In 14 pazienti abbiamo potuto studiare la malattia minima anche nel sangue periferico. 10/14 sono risultati positivi con valori di Ct in un range da 35 a 38, indipendentemente dalla positivit\ue0 midollare. Conclusioni. Questi risultati preliminari suggeriscono che il coinvolgimento midollare alla diagnosi \ue8 discretamente frequente in pazienti con RMS e che la MMR pu\uf2 essere rilevata, anche se a livelli molto bassi, nel sangue periferico indipendentemente dall\u2019infiltrazione midollare

Results of a prospective minimal disseminated disease study in human rhabdomyosarcoma using three different molecular markers

BACKGROUND: Rhabdomyosarcoma (RMS) has 2 major histologic subtypes: alveolar (ARMS) and embryonal (ERMS). ARMS is more aggressive and prone to distant tumor dissemination, whereas ERMS tends to expand and recur locally. Little information is available on bone marrow involvement by RMS. METHODS: We determined the sensitivity and specificity of MyoD1, myogenin, and PAX-FKHR transcripts as RMS markers and used them to study prospectively by reverse-transcriptase polymerase chain reaction (RT-PCR) a series of consecutive unselected RMS patients enrolled in the Italian Association of Pediatric Hematology and Oncology national trial. Prevalence of minimal disseminated disease (MDD) and its response kinetics to chemotherapy were assessed. RESULTS: MyoD1 and myogenin were specifically associated with RMS, independently of histologic subtype, whereas PAX3/7-FKHR transcripts were expressed only in ARMS. Sensitivity was higher for MyoD1 compared with myogenin and PAX-FKHR. Out of a cohort of 40 patients, MDD positivity was limited to ARMS, with the sole exception of 1 ERMS. Prevalence of MDD positivity increased when a real-time polymerase chain reaction approach was used on a subgroup of patients. RT-PCR was more sensitive than microscopic examination of bone marrow biopsies. The study of the response kinetics of MDD showed that in approximately half of the cases, bone marrow was cleared of disease after 1 cycle of chemotherapy. CONCLUSIONS: MyoD1 and myogenin transcripts can be used as tumor markers for MDD assessment in virtually all RMS cases, whereas PAX-FKHR is specific for ARMS. Sensitivity of RT-PCR methods was superior compared with standard morphologic assays. Our study suggests that bone marrow involvement is more common in ARMS compared with ERMS, and that MDD can be often cleared by the initial chemotherapy cycles