15 research outputs found
How to manage symptoms in pediatric cancer patients enrolled in clinical trials? A review of principal patient-reported outcome screening tools
Background: In adult oncology, the practice of tracking symptoms and toxicities using patient-reported outcomes (PROs) has increased and correlates with increased survival. In contrast, symptom monitoring using PROs is not common in pediatric oncology. Only in the last couple of years attention has also been paid to the patient’s perception in pediatrics and listening to the voice of children and to making them participate in the treatment.
Methods:Â A comprehensive literature search was conducted in MEDLINE/PubMed and PsycINFO to identify relevant articles published through December 2022.
Results:Â From 58 non-duplicate articles, 33 met our eligibility criteria. Of these, 17 were used in clinical trials.
Conclusions:Â The dissemination and use of these tools will therefore have surprising repercussions on the control of pain and physical symptoms of small patients as well as on physical and psychological aspects. The administration and use of the PROs ensures optimal use of the drugs currently present in clinical trials by researcher and nurse and aims at a safer and more controlled approval of new drugs
The PedAL/EuPAL Project:A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia
The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40–50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children’s Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript.</p
The PedAL/EuPAL Project:A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia
The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40–50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children’s Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript.</p
Targeting type-2 metabotropic glutamate receptors to protect vulnerable hippocampal neurons against ischemic damage
Gynecomastia after euthyroidism restoration in a patient with type 1 diabetes and Graves’ disease
In patients with autoimmune disease, gynecomastia should not be considered as 1 of the first signs of hyperthyroidism, rather it is a breast pathology that can be present even when euthyroidism restoration is achieved. It is unknown whether the autoimmune nature of thyroid disorders or simply the hyperthyroidism effects breast changes
Off-label and compassionate use of targeted anticancer therapies: The experience of an Italian pediatric cancer center
Immune-mediated cytopenias (IMCs) after HSCT for pediatric non-malignant disorders: epidemiology, risk factors, pathogenesis, and treatment
Hematopoietic stem cell transplantation (HSCT) represents a curative option for pediatric patients affected by malignant and non-malignant disorders. Several complications may arise during the post-transplantation period, including immune-mediated disorders. Immune-mediated cytopenias (IMCs) account for up to 22% of pediatric HSCT complications, representing an important cause of morbidity and mortality post-HSCT. So far, their pathogenesis is not well-understood, and their management may be very challenging. Further, most patients are refractory to first-line treatment which is based on high-dose intravenous steroids, immunoglobulin, and the monoclonal anti-CD20 antibody — rituximab. No clear consensus has been reached for second- and third-line therapeutic options. Conclusion: We reviewed the epidemiology, risk factors, pathogenesis, and treatment of IMCs, aiming to offer a deeper understanding of these complications as a guide to improving the management of these fragile patients and a cue for the design of tailored clinical trials.What is Known:• IMCs arising in the post-HSCT setting represent a rare but potentially life-threatening complication. Younger patients affected by non-malignant disorders are at the greatest risk of IMCs arising after HSCT. Corticosteroids, intravenous immunoglobulin, and rituximab represent the undiscussed first-line therapeutic approach.What is New:• This review highlitghts how children present unique risk factors for post HSCT IMCs, which are the result of the complex relationship between the immaturity of their infantile immune system and all the perturbing agents and factors which characterize the post-HSCT setting. Future efforts are warranted to establish the best option for refractory patients, for whom a standard and validated approach is not currently available. Among new agents, ibrutinib or bortezomib and fostamatinib or low-dose IL-2 could represent a good therapeutic option for patients with graft-versus-host disease and hemolytic anemia or graft-versus-host disease and thrombocytopenia, respectively
Questões éticas do cotidiano profissional e a formação do enfermeiro
Estudo de natureza qualitativa que objetivou conhecer as percepções das enfermeiras sobre como vivenciam
os principais problemas éticos do cotidiano do trabalho e se a formação profissional recebida na graduação mostrou-se
suficiente para o enfrentamento destes problemas. Os dados foram coletados entre março e abril de 2009, junto a cinco
enfermeiras de uma unidade de terapia intensiva de um hospital universitário do Rio Grande do Sul, por meio de observações
livres seguidas de entrevista semiestruturada. Utilizou-se a técnica de análise textual qualitativa no tratamento dos
dados. Dos resultados emergiram duas categorias: a dimensão ética do cuidado e na formação profissional. Mostrou-se
necessária uma reflexão mais profunda sobre o cotidiano do trabalho ainda na graduação, fortalecendo as futuras enfermeiras
para o enfrentamento do exercÃcio profissional. Paralelamente, precisa-se priorizar a construção de espaços para
a reflexão e discussão coletiva dos profissionais atuantes, identificando e construindo novas maneiras de atuar eticamente.This qualitative study aimed to learn nurses’ perceptions of how they experience the main ethical problems
of their day-to-day work and whether the professional training they received in their undergraduate studies has proved
sufficient to meet these problems. Data were collected from March to April 2009 by free observation followed by semistructured
interviews of five nurses at a university hospital intensive care unit (ICU) in Rio Grande do Sul, and were treated
using the technique of qualitative textual analysis. Two categories emerged from the results: the ethical dimension in care
and in professional training. There was found to be a need for undergraduate studies to consider in greater depth the dayto-
day experience of nursing work, so as to strengthen future nurses to exercise the profession. There is a parallel need to
prioritize construction of settings where practicing nurses can think and discuss together with a view to identifying and
building new ways to act ethically.Estudio de naturaleza cualitativa que objetivó conocer las percepciones de las enfermeras, acerca de como
vivencian los principales problemas éticos del trabajo diario y sà la formación profesional recibida en el pregrado se ha
mostrado suficiente para hacer frente a estos problemas. Los datos fueron recolectados entre marzo y abril de 2009, con
cinco enfermeras en una unidad de cuidados intensivos (UCI) de un hospital universitario de RÃo Grande do Sul-Brasil, por
medio de observaciones libres, seguidas de entrevista semiestructurada. Se utilizó la técnica de análisis textual cualitativa
en el tratamiento de los datos. Los resultados destacan dos categorÃas: dimensión ética de la atención y en la formación
profesional. Se ha mostrado necesaria una reflexión más profunda sobre el trabajo diario aun en los estudios de graduación,
fortaleciendo las futuras enfermeras para enfrentar el futuro de la profesión. Al mismo tiempo, es necesario dar prioridad a
la construcción de espacios de reflexión y discusión colectiva de los profesionales actuantes, identificando y construyendo
nuevas formas de actuar éticamente
Cuestiones éticas del cotidiano profesional y la formación del enfermero
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Previous issue date: 2011Estudo de natureza qualitativa que objetivou conhecer as percepções das enfermeiras sobre como vivenciam
os principais problemas éticos do cotidiano do trabalho e se a formação profissional recebida na graduação mostrou-se
suficiente para o enfrentamento destes problemas. Os dados foram coletados entre março e abril de 2009, junto a cinco
enfermeiras de uma unidade de terapia intensiva de um hospital universitário do Rio Grande do Sul, por meio de observações
livres seguidas de entrevista semiestruturada. Utilizou-se a técnica de análise textual qualitativa no tratamento dos
dados. Dos resultados emergiram duas categorias: a dimensão ética do cuidado e na formação profissional. Mostrou-se
necessária uma reflexão mais profunda sobre o cotidiano do trabalho ainda na graduação, fortalecendo as futuras enfermeiras
para o enfrentamento do exercÃcio profissional. Paralelamente, precisa-se priorizar a construção de espaços para
a reflexão e discussão coletiva dos profissionais atuantes, identificando e construindo novas maneiras de atuar eticamente.This qualitative study aimed to learn nurses’ perceptions of how they experience the main ethical problems
of their day-to-day work and whether the professional training they received in their undergraduate studies has proved
sufficient to meet these problems. Data were collected from March to April 2009 by free observation followed by semistructured
interviews of five nurses at a university hospital intensive care unit (ICU) in Rio Grande do Sul, and were treated
using the technique of qualitative textual analysis. Two categories emerged from the results: the ethical dimension in care
and in professional training. There was found to be a need for undergraduate studies to consider in greater depth the dayto-
day experience of nursing work, so as to strengthen future nurses to exercise the profession. There is a parallel need to
prioritize construction of settings where practicing nurses can think and discuss together with a view to identifying and
building new ways to act ethically.Estudio de naturaleza cualitativa que objetivó conocer las percepciones de las enfermeras, acerca de como
vivencian los principales problemas éticos del trabajo diario y sà la formación profesional recibida en el pregrado se ha
mostrado suficiente para hacer frente a estos problemas. Los datos fueron recolectados entre marzo y abril de 2009, con
cinco enfermeras en una unidad de cuidados intensivos (UCI) de un hospital universitario de RÃo Grande do Sul-Brasil, por
medio de observaciones libres, seguidas de entrevista semiestructurada. Se utilizó la técnica de análisis textual cualitativa
en el tratamiento de los datos. Los resultados destacan dos categorÃas: dimensión ética de la atención y en la formación
profesional. Se ha mostrado necesaria una reflexión más profunda sobre el trabajo diario aun en los estudios de graduación,
fortaleciendo las futuras enfermeras para enfrentar el futuro de la profesión. Al mismo tiempo, es necesario dar prioridad a
la construcción de espacios de reflexión y discusión colectiva de los profesionales actuantes, identificando y construyendo
nuevas formas de actuar éticamente