Difficulties in decision making during the final phase of life in a boy with spinal muscular atrophy [Dificultades en la toma de decisiones en la fase final de la vida de un niño con atrofia muscular espinal]
Quiroga Cantero E.,Oncologia Pediatrica |
Madruga Garrido M.,Neurologia Pediatrica |
Falcon Neyra M.,Medicina Interna Pediatrica |
Morillo Gutierrez B.,Medicina Interna Pediatrica
The majority of the dilemmas in palliative care occur during the final moments of life. An additional and important problem occurs in paediatrics where in the majority of cases the principles of autonomy cannot be accounted due to the young age of the child. This leaves the decision making to its caretakers, who together with the paediatricians aim to reach an agreement which must be in the best interest of the patient. Here we present the ethical difficulties in the management of a seven months old infant suffering from a progressive neuromuscular disease during the final stage of life. Source
Alaggio R.,University of Padua |
Turrini R.,Veneto Institute of Oncology IOV IRCCS |
Boldrin D.,Veneto Institute of Oncology IOV IRCCS |
Merlo A.,Veneto Institute of Oncology IOV IRCCS |
And 11 more authors.
Malignant peripheral nerve sheath tumors (MPNST) are very aggressive malignancies comprising approximately 5-10% of all soft tissue sarcomas. In this study, we focused on pediatric MPNST arising in the first 2 decades of life, as they represent one the most frequent non-rhabdomyosarcomatous soft tissue sarcomas in children. In MPNST, several genetic alterations affect the chromosomal region 17q encompassing the BIRC5/SURVIVIN gene. As cancer-specific expression of survivin has been found to be an effective marker for cancer detection and outcome prediction, we analyzed survivin expression in 35 tumor samples derived from young patients affected by sporadic and neurofibromatosis type 1-associated MPNST. Survivin mRNA and protein expression were assessed by Real-Time PCR and immunohistochemical staining, respectively, while gene amplification was analyzed by FISH. Data were correlated with the clinicopathological characteristics of patients. Survivin mRNA was overexpressed in pediatric MPNST and associated to a copy number gain of BIRC5; furthermore, increased levels of transcripts correlated with a higher FNCLCC tumor grade (grade 1 and 2 vs. 3, p = 0.0067), and with a lower survival probability (Log-rank test, p = 0.0038). Overall, these data support the concept that survivin can be regarded as a useful prognostic marker for pediatric MPNST and a promising target for therapeutic interventions. © 2013 Alaggio et al. Source
Daikeler T.,University Paris Diderot |
Daikeler T.,University of Basel |
Labopin M.,CEREST TC European Group for Blood and Marrow Transplantation |
Ruggeri A.,University Paris Diderot |
And 24 more authors.
To describe the incidence, risk factors, and treatment of autoimmune diseases (ADs) occurring after cord blood transplantation (CBT), we analyzed both CBT recipients reported to EUROCORD who had developed at least 1 new AD and those who had not. Fifty-two of 726 reported patients developed at least 1 AD within 212 days (range, 27-4267) after CBT. Cumulative incidence of ADs after CBT was 5.0% ± 1% at 1 year and 6.6% ± 1% at 5 years. Patients developing ADs were younger and had more nonmalignant diseases (P < .001). ADs target hematopoietic (autoimmune hemolytic anemia, n = 20; Evans syndrome, n = 9; autoimmune thrombocytopenia, n = 11; and immune neutropenia, n = 1) and other tissues (thyroiditis, n = 3; psoriasis, n = 2; Graves disease, n = 1; membranous glomerulonephritis, n = 2; rheumatoid arthritis, n = 1; ulcerative colitis, n = 1; and systemic lupus erythematosus, n = 1). Four patients developed 2 ADs (3 cases of immune thrombocytopenia followed by autoimmune hemolytic anemia and 1 Evans syndrome with rheumatoid arthritis). By multivariate analysis, the main risk factor for developing an AD was nonmalignant disease as an indication for CBT (P = .0001). Hematologic ADs were most often treated with steroids, rituximab, and cyclosporine. With a median follow-up of 26 months (range, 2-91), 6 of 52 patients died as a consequence of ADs. We conclude that CBT may be followed by potentially life-threatening, mainly hematologic ADs. © 2013 by The American Society of Hematology. Source
Allogeneic Hematopoietic Stem Cell Transplantation for Philadelphia-Positive Acute Lymphoblastic Leukemia in Children and Adolescents: A Retrospective Multicenter Study of the Italian Association of Pediatric Hematology and Oncology (AIEOP)
Fagioli F.,Oncoematologia Pediatrica e Centro Trapianti |
Zecca M.,Oncoematologia Pediatrica |
Rognoni C.,University of Pavia |
Balduzzi A.,University of Milan Bicocca |
And 8 more authors.
Biology of Blood and Marrow Transplantation
Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) still represents a major challenge. We report the experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP) with allogeneic hematopoietic stem cell transplantation (HSCT) in children with Ph+ ALL from 1990 to 2008. Sixty-nine patients received HSCT from either a related (37, 54%) or an unrelated (32, 46%) donor. Twenty-five patients (36%) underwent transplantation before 2000 and 44 (64%) after 2000. Twenty-three patients (33%) received Imatinib mesylate treatment before HSCT and seven (10%) after HSCT. After a median follow-up of 56 months, the overall survival (OS) probability was 51% (95% confidence interval [CI], 38-63), the leukemia-free survival (LFS) was 47% (95% CI, 34-59), transplantation-related mortality (TRM) was 17% (95% CI, 10-30), and relapse incidence (RI) was 36% (95% CI, 26-50). Transplantation in first complete remission, female gender, and lower WBC count at diagnosis were associated with a better LFS in both univariate and multivariate analyses. Patients with p210 transcript had a trend for a worse prognosis compared with those who had the p190 transcript. Our series confirms the role of HSCT in the eradication of Ph+ ALL. Early HSCT is recommended once morphologic remission is obtained. © 2012 American Society for Blood and Marrow Transplantation. Source
Farruggia P.,Pediatric Hematology and Oncology Unit |
Puccio G.,University of Palermo |
Sala A.,University of Milan Bicocca |
Todesco A.,University of Padua |
And 14 more authors.
European Journal of Cancer
Background Many biological and inflammatory markers have been proposed as having a prognostic value at diagnosis of Hodgkin lymphoma (HL), but very few have been validated in paediatric patients. We explored the significance of these markers in a large population of 769 affected children. Patients and methods By using the database of patients enrolled in A.I.E.O.P. (Associazione Italiana di Emato-Oncologia Pediatrica) trial LH2004 for paediatric HL, we identified 769 consecutive patients treated with curative intent from 1st June 2004 to 1st April 2014 with ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine), or hybrid COPP/ABV (cyclophosphamide, vincristine, prednisone, procarbazine, doxorubicin, bleomycin and vinblastine) regimens. Results On multivariate analysis with categorical forms, the 5-year freedom from progression survival was significantly lower in patients with stage IV or elevated value of platelets, eosinophils and ferritin at diagnosis. Furthermore, stage IV and eosinophils seem to maintain their predictive value independently of interim (after IV cycles of chemotherapy) positron emission tomography. Conclusion Using the combination of four simple markers such as stage IV and elevated levels of platelets, ferritin and eosinophils, it is possible to classify the patients into subgroups with very different outcomes. © 2015 Elsevier Ltd. Source