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J Neurooncol ; 145(1): 177-184, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31522324


INTRODUCTION: Diffuse intrinsic pontine glioma (DIPG) is a rare clinically, neuro-radiologically, and molecularly defined malignancy of the brainstem with a median overall survival of approximately 11 months. Our aim is to evaluate the current tendency for its treatment in Europe in order to develop (inter)national consensus guidelines. METHODS: Healthcare professionals specialized in DIPG were asked to fill in an online survey with questions regarding usual treatment strategies at diagnosis and at disease progression in their countries and/or their centers, respectively. RESULTS: Seventy-four healthcare professionals responded to the survey, of which 87.8% were pediatric oncologists. Only 13.5% of the respondents biopsy all of their patients, 41.9% biopsy their patients infrequently. More than half of the respondents (54.1%) treated their patients with radiotherapy only at diagnosis, whereas 44.6% preferred radiotherapy combined with chemotherapy. When the disease progresses, treatment strategies became even more diverse, and the tendency for no treatment increased from 1.4% at diagnosis to 77.0% after second progression. 36.5% of the healthcare professionals treat children younger than 3 years differently than older children at diagnosis. This percentage decreased, when the disease progresses. Most of the participants (51.4%) included less than 25% of their patients in clinical trials. CONCLUSION: This survey demonstrates a large heterogeneity of treatment regimens, especially at disease progression. We emphasize the need for international consensus guidelines for the treatment of DIPG, possible by more collaborative clinical trials.

J Pediatr Hematol Oncol ; 41(7): 525-531, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31274668


This phase 2, multicenter, open-label trial investigated the safety and tolerability of tbo-filgrastim in pediatric patients receiving myelosuppressive chemotherapy. In total, 50 patients 1 month to below 16 years of age with solid tumors without bone marrow involvement were stratified into 3 age groups (2 infants, 30 children, 18 adolescents) and prophylactically administered tbo-filgrastim 5 µg/kg body weight once daily subcutaneously. The administration started after the last chemotherapy treatment in week 1 of the first cycle and continued until the expected neutrophil nadir had passed, and the neutrophil count had recovered to 2.0×10/L. The primary endpoint was safety and tolerability of tbo-filgrastim; secondary endpoints included efficacy. The mean (SD) number of doses administered was 9.2 (2.83) in children and 7.3 (1.88) in adolescents. Serious treatment-emergent adverse events were reported in 24% of patients; the most common were febrile neutropenia (FN) (12%), anemia (8%), and thrombocytopenia (8%). Nine patients (18%) experienced mild treatment-related treatment-emergent adverse events; the most common were musculoskeletal and connective tissue disorders (8%). No deaths or withdrawals occurred. The incidence of severe neutropenia (SN) was 52% and the mean (SD) duration of SN was 1.8 (2.21) days; FN incidence was 26%. A daily dose of tbo-filgrastim 5 µg/kg body weight administered to pediatric patients demonstrated a safety profile consistent with the safety profile in adult patients. The incidence of FN was on the lower end of the range reported in the literature and the SN results provide supportive data on the efficacy of tbo-filgrastim in pediatric patients.

J Med Ethics ; 42(12): 757-761, 2016 12.
Artigo em Inglês | MEDLINE | ID: mdl-27697792


Communication about diagnosis and medical treatment for children suffering from life-threatening illnesses is complex. It is a primary step in involving underage patients and families in care and lays the foundation for obtaining parental permission and patient assent for treatment. In practice child participation in care is often difficult to obtain due to patients' different and sometimes fluctuating preferences, but also parents' protective strategies. Physicians may be susceptible to parental wishes to limit information and feel uncomfortable discussing issues related to uncertainty of cure with patients. A qualitative study in Romanian paediatric oncology units was conducted to explore children's involvement from the perspectives of parents and oncologists. Interviews with participants discussed 18 patient cases. Data were transcribed and thematic analysis was used to interpret and mine patients' involvement during treatment. Different facets of patient participation were identified: restricting, widening and enhancing involvement. A fourth category, unintentional involvement, occurred for all patients due to children's observations during long-term hospitalisations and access to Internet. Uncertainty overarched parental attitudes regarding the extent to which children should be included. Physicians usually complied with parental wishes to limit involvement, but together with parents involved patients at least in a practical way. Adults' protective attitude may backfire, as adolescents' online searches often expose patients to worse-case scenarios. Further research should acknowledge the hazards of restricted diagnosis disclosure and develop clinician tools to support families in communicating with patients. This should be paralleled by physician efforts to elicit patients' needs regarding participation.

Comunicação , Neoplasias , Relações Pais-Filho , Pais , Participação do Paciente , Preferência do Paciente , Relações Médico-Paciente , Adolescente , Comportamento do Adolescente , Atitude , Criança , Tomada de Decisões , Ética Médica , Feminino , Humanos , Intenção , Internet , Masculino , Oncologia , Neoplasias/terapia , Pediatria , Pesquisa Qualitativa , Romênia , Inquéritos e Questionários
Pediatr Blood Cancer ; 62(12): 2177-82, 2015 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-26207515


BACKGROUND: Professional guidelines encourage physicians to provide children with as much information regarding their health as deemed developmentally and emotionally appropriate. However, empirical research indicates that in clinical practice, an open discussion with children about cancer is often lacking. This study explores impeding factors to and possible strategies for open communication of cancer diagnosis to children from the perspectives of parents and physicians. PROCEDURE: Semi-structured interviews were conducted with 18 parents of children with cancer and 10 treating oncologists. The patient sample was obtained from three pediatric units in Romania. Interviews were transcribed verbatim and interpreted using thematic analysis. Inductive open-coding procedures identified participants' accounts regarding their experiences with cancer diagnosis and treatment. Final themes were selected by grouping codes that formed a pattern in the data. RESULTS: An interplay of mainly three different factors-information overload and emotional turmoil, lack of knowledge and skills for disclosing the diagnosis, and assumptions about burdening the child when discussing cancer-restricted parent-patient communication and subsequently affected physician-patient exchanges. Oncologists recommended open communication at diagnosis, but left the final decision to the parents. They adapted their communication style with patients to parents' preference. CONCLUSIONS: Although physicians need to respect the wishes of children's legal representatives, they also have a duty to promote patients' best interests. We recommend that physicians employ a proactive stance in ensuring that children with cancer are appropriately informed about their diagnosis. In case of parents' arduous objections to full disclosure, an ethical consultation should be considered.

Neoplasias/psicologia , Notificação aos Pais , Relações Profissional-Paciente , Revelação da Verdade , Adolescente , Adulto , Criança , Humanos , Masculino , Romênia
Rom J Intern Med ; 41(3): 323-8, 2003.
Artigo em Inglês | MEDLINE | ID: mdl-15526516


BACKGROUND: Granulomas are encountered in 1-2% of biopsies performed in various hematological and non-hematological diseases. Almost 50% of bone marrow granulomas are associated with infections and 25% with hematologic disorders, especially lymphoma and multiple myeloma. Toxoplasmosis is reported to induce granulomas in bone marrow inmunosuppressed patients. On the other side, long-term unexplained remissions after conventional treatment in multiple myeloma were mentioned in up to 10% of cases. CASE REPORT: A 56-years-old female patient was diagnosed with IgG(kappa) multiple myeloma in 1992. After 5 years, being still in complete remission, frequent bone marrow epithelioid non-caseating granulomas were noticed in biopsy, without clinical symptomatology or modifications of routine paraclinical examinations. The history revealed no treatments with antiarrhythmic, antihypertensive, anticonvulsivants or nonsteroid antiinflammatory drugs. The serologic tests for other infections or systemic diseases known to induce granulomas were negative, except those for toxoplasma gondii IgG. The treatment with azithromycine and pyrimethamine induced the disappearance of granulomas, simultaneously with an important decrease of anti-toxoplasma IgG antibodies titer. CONCLUSIONS: The bone marrow granulomas provide a valuable histologic clue to opportunistic infections and the bone marrow biopsy is useful for their diagnosis. In the specific case of toxoplasmosis, a recently proposed treatment with azithromycin induced the resolution of the granulomas. Due to the usual lack of specificity of the most bone marrow granulomas, a broad and long-term clinical, histopatological and serological follow-up to establish the etiology should be performed.

Doenças da Medula Óssea/etiologia , Granuloma/etiologia , Mieloma Múltiplo/complicações , Toxoplasmose/complicações , Antiprotozoários/uso terapêutico , Azitromicina/uso terapêutico , Doenças da Medula Óssea/tratamento farmacológico , Doenças da Medula Óssea/patologia , Exame de Medula Óssea , Feminino , Granuloma/tratamento farmacológico , Granuloma/patologia , Humanos , Pessoa de Meia-Idade , Pirimetamina/uso terapêutico , Indução de Remissão , Toxoplasmose/tratamento farmacológico , Toxoplasmose/patologia , Resultado do Tratamento