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1.
Pediatr Blood Cancer ; 71(7): e30990, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38605511

RESUMO

BACKGROUND: Central venous catheter (CVC)-related complications remain a significant cause of morbidity in pediatric hematology-oncology. We prospectively surveyed the incidence of CVC-related complications in children with hematologic-oncologic diseases. PROCEDURE: Five-hundred-eighty-one CVCs were inserted in 421 patients from January 2010 to June 2022 (153,731 CVC days observation; follow-up data up to December 31, 2022). RESULTS: Overall, 671 complications were recorded (4.365/1000 CVC days): 49.7% malfunctions (1.88/1000 CVC days, 4.8% of CVC early removals), 23.9% bacteremia (0.90/1000, 15.1%), 19.6% mechanical complications (0.74/1000, 70.2%), 20.1% localized infections (0.76/1000, 17.1%), 0.5% thrombosis (0.02/1000, 33.3%). At multivariate analysis, risk factors for malfunction were Broviac-Hickman type of CVC (hazard ratio [HR] 2.5) or Port-a-cath (HR 3.4) or Proline (HR 4.3), p < .0001; for bacteremia double-lumen CVC (HR 3.2, p < .0001); for mechanical complications age at CVC insertion under median (HR 4.5, p < .0001) and Broviac-Hickman (HR 1.6) or Proline (HR 2.7), p = .01; finally for localized infections Broviac-Hickman (HR 2.9) or Proline (HR 4.4), p = .0001. The 2-year cumulative incidence of premature removal was 23.5%, and risk factors were age at CVC insertion under median (HR 2.4, p < .0001), Broviac-Hickman (HR 2.3) or Proline (HR 4.2), p < .0001. CONCLUSIONS: Premature removal occurs in approximately 20%-25% of long-term CVCs. A surveillance program has a fundamental role in identifying the risk factors for CVC complications and the areas of intervention to improve CVC management.


Assuntos
Infecções Relacionadas a Cateter , Cateterismo Venoso Central , Cateteres Venosos Centrais , Neoplasias Hematológicas , Humanos , Feminino , Criança , Masculino , Estudos Prospectivos , Pré-Escolar , Cateteres Venosos Centrais/efeitos adversos , Adolescente , Lactente , Infecções Relacionadas a Cateter/epidemiologia , Infecções Relacionadas a Cateter/etiologia , Cateterismo Venoso Central/efeitos adversos , Neoplasias Hematológicas/terapia , Seguimentos , Fatores de Risco , Bacteriemia/etiologia , Bacteriemia/epidemiologia , Incidência , Prognóstico
3.
Clin Hematol Int ; 5(2-3): 130-138, 2023 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-37072555

RESUMO

The treatment of pediatric patients with refractory or relapsed anaplastic large cell lymphoma (ALCL) is still a major challenge. In addition to conventional chemotherapy and stem cell transplantation, new therapeutic options such as anti-CD30 drugs and anaplastic lymphoma kinase (ALK) inhibitors have been recently introduced in this setting. Among ALK inhibitors, only the first-generation molecule crizotinib is approved for pediatric use, while second-generation molecules, such as brigatinib, are still under investigation. Here we report the case of a 13-year-old boy diagnosed with stage IV ALCL, refractory to first-line conventional chemotherapy and second-line therapy with the anti CD30 antibody-drug conjugate brentuximab-vedotin, who finally achieved remission after a combination of conventional high-dose chemotherapy and the second-generation ALK inhibitor brigatinib. The latter was chosen for its ability to penetrate through the blood-brain barrier, due to the persistent involvement of the patient's cerebral nervous system. The remission was then consolidated with an allogeneic hematopoietic stem cell transplantation (HSCT) from an unrelated donor using myeloablative conditioning with total body irradiation. At 24 months after HSCT, the patient is in complete remission, alive and well. An updated review regarding the use of ALK inhibitors in ALCL patients is provided.

4.
J Clin Med ; 11(3)2022 Feb 08.
Artigo em Inglês | MEDLINE | ID: mdl-35160334

RESUMO

Bloodstream infections (BSIs) after chemotherapy or hematopoietic stem cell transplantation (HSCT) are a leading cause of morbidity and mortality. Data on 154 BSIs that occurred in 111 onco-hematological patients (57 hematological malignancies, 28 solid tumors, and 26 non-malignant hematological diseases) were retrospectively collected and analyzed. Monomicrobial Gram-positive (GP), Gram-negative (GN), and fungal BSIs accounted for 50% (77/154), 38.3% (59/144), and 3.2% (5/154) of all episodes. Polymicrobial infections were 7.8% (12/154), while mixed bacterial-fungal infections were 0.6% (1/154). The most frequent GN isolates were Escherichia coli (46.9%), followed by Pseudomonas aeruginosa (21.9%), Klebsiella species (18.8%), and Enterobacter species (6.3%). Overall, 18.8% (12/64) of GN organisms were multidrug-resistant (seven Escherichia coli, three Klebsiella pneumoniae, and two Enterobacter cloacae), whereas GP resistance to glycopeptides was observed in 1% (1/97). Initial empirical antibiotic therapy was deemed inappropriate in 12.3% of BSIs (19/154). The 30-day mortality was 7.1% (11/154), while the bacteremia-attributable mortality was 3.9% (6/154). In multivariate analysis, septic shock was significantly associated with 30-day mortality (p = 0.0001). Attentive analysis of epidemiology and continuous microbiological surveillance are essential for the appropriate treatment of bacterial infections in pediatric onco-hematological patients.

5.
Hematol Rep ; 13(1): 8847, 2021 Mar 05.
Artigo em Inglês | MEDLINE | ID: mdl-33747412

RESUMO

Long-term survival for acute lymphoblastic leukemia (ALL) in children improved over the last three decades up to 80-90% of affected patients. Consequently, the quality of life of survivors has become increasingly important. This study analyses the clinical features and outcome of 119 children with ALL, focusing on the quality of long-term survival in a subset of 22 patients over 18 years of age. Among this group, the 10-year event-free survival and overall survival were 83.1% (C.I. 74.0-89.2) and 88.4% (C.I. 80.9-93.1), respectively. Treatment related long-term medical complications were reported only in 2 patients (9.1%). Secondary school was completed successfully in 20 of 22 patients (89.9%). The remaining 2 patients were still attending at the time of the analysis. In conclusion, current treatment for ALL is well tolerated and does not compromise significantly the quality of life of survivors.

6.
Mediterr J Hematol Infect Dis ; 12(1): e2020079, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33194153

RESUMO

BACKGROUND: Invasive mucormycosis is a very aggressive fungal disease among immunocompromised pediatric patients caused by saprophytic fungi that belong to the order of the Mucorales. CASE REPORT: We describe a case of of Lichtheimia corymbifera infection in a 15-year-old child with B-cell-Non-Hodgkin Lymphoma (B-NHL) involving lung, kidney and thyroid that initially was diagnosed as probable aspergillosis delaying the effective therapy for mucormycosis. CONCLUSIONS: This case showed that also the intensive chemotherapy for B-NHL may represent a risk factor for mucormycosis infection. Liposomal amphotericin B and surgery remain the key tools for the successful treatment of this aggressive disease.

7.
Front Pediatr ; 8: 580963, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33178652

RESUMO

Lipopolysaccharide responsive beige-like anchor protein (LRBA) deficiency is a primary immunodeficiency disorder (PID) that can cause a common variable immunodeficiency (CVID)-like disease. The typical features of the disease are autoimmunity, chronic diarrhea, and hypogammaglobulinemia. Neurological complications are also reported in patients affected by LRBA deficiency. We describe a 7-year old female with an acute cervical longitudinally extensive transverse myelitis (LETM) as a feature of LRBA deficiency. This is the first case of LETM associated with LRBA deficiency described in literature.

8.
Mediterr J Hematol Infect Dis ; 12(1): e2020002, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-31934312

RESUMO

BACKGROUND: Chronic graft versus host disease (cGVHD) occurs in 20-30% of paediatric patients receiving haemopoietic stem cell transplantation (HSCT). Neuromuscular disorders such as polymyositis are considered a rare and distinctive but non-diagnostic manifestation of cGVHD and, in the absence of other characteristic signs and symptoms, biopsy is highly recommended to exclude other causes. CASE REPORT: We report a case of a 17-months-old child affected by hemophagocytic lymphohistiocytosis who underwent a matched unrelated donor haematopoietic stem cell transplantation (HSCT). She developed severe cGVHD-related polymyositis that was successfully treated with high-dose steroid therapy, rituximab and sirolimus. CONCLUSIONS: This is the first case of cGVHD-related-polymyositis described in a pediatric patient which was successfully treated with rituximab.

9.
Front Pediatr ; 7: 51, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-30863741

RESUMO

The polyglandular autoimmune syndrome type I is a rare hereditary autosomal recessive disease. We describe a child with the classic triad of the disease and her sister with pure red cell aplasia and cerebellar hypoplasia. The latter received two haematopoietic stem cell transplantations, complicated by an acute disseminated encephalomyelitis.

10.
Mediterr J Hematol Infect Dis ; 10(1): e2018043, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30002799

RESUMO

BACKGROUND: Kaposiform Hemangioendothelioma (KHE) is a rare vascular tumour of the infancy and the first decade of life. It is locally aggressive and potentially life threatening when associated with consumptive coagulopathy, known as Kasabach-Merritt syndrome (KMS). No consensus or guideline for the therapy has been reached because of the lack of prospective trials, and the different standard care suggestions are based on retrospective case series. CASE REPORT: We report the case of a 9-month-old male with KHE and KMS in which the initial response, obtained with prednisone and vincristine, was subsequently consolidated and strengthened by long-term treatment with sirolimus, a mTOR inhibitor. A summary of the published data is presented as well. CONCLUSIONS: The inhibition of mTOR pathway represents the most important therapeutic innovation introduced in the last few years for KHE. Our case shows the effectiveness and good tolerance of long-term therapy with sirolimus.

11.
Pediatr Blood Cancer ; 65(6): e26963, 2018 06.
Artigo em Inglês | MEDLINE | ID: mdl-29350496

RESUMO

We describe three cases of sinusoidal obstruction syndrome/venoocclusive disease (SOS) in pediatric patients with acute lymphoblastic leukemia (ALL). All three episodes occurred during or just after the induction or reinduction phase of treatment based on prednisone/dexamethasone, vincristine, daunorubicin, and pegylated-l-asparaginase. SOS episodes were categorized as mild/moderate and resolved in 7, 10, and 16 days using supportive measures or defibrotide therapy. In all three episodes, the clinical diagnosis of SOS was associated with a significant increase in plasminogen-activator inhibitor-1 (PAI-1) that reduced with patient clinical improvement. PAI-1 warrants study as a diagnostic marker for SOS in ALL.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Hepatopatia Veno-Oclusiva/diagnóstico , Inibidor 1 de Ativador de Plasminogênio/metabolismo , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Adolescente , Criança , Pré-Escolar , Feminino , Hepatopatia Veno-Oclusiva/induzido quimicamente , Hepatopatia Veno-Oclusiva/metabolismo , Humanos , Masculino , Prognóstico
12.
Front Immunol ; 9: 2767, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30692987

RESUMO

Adenosine deaminase 2 (ADA2) deficiency is an auto-inflammatory disease due to mutations in cat eye syndrome chromosome region candidate 1 (CECR1) gene, currently named ADA2. The disease has a wide clinical spectrum encompassing early-onset vasculopathy (targeting skin, gut and central nervous system), recurrent fever, immunodeficiency and bone marrow dysfunction. Different therapeutic options have been proposed in literature, but only steroids and anti-cytokine monoclonal antibodies (such as tumor necrosis factor inhibitor) proved to be effective. If a suitable donor is available, hematopoietic stem cell transplantation (HSCT) could be curative. Here we describe a case of ADA2 deficiency in a 4-year-old Caucasian girl. The patient was initially classified as autoimmune neutropenia and then she evolved toward an autoimmune lymphoproliferative syndrome (ALPS)-like phenotype. The diagnosis of ALPS became uncertain due to atypical clinical features and normal FAS-induced apoptosis test. She was treated with G-CSF first and subsequently with immunosuppressive drugs without improvement. Only HSCT from a 9/10 HLA-matched unrelated donor, following myeloablative conditioning, completely solved the clinical signs related to ADA2 deficiency. Early diagnosis in cases presenting with hematological manifestations, rather than classical vasculopathy, allows the patients to promptly undergo HSCT and avoid more severe evolution. Finally, in similar cases highly suspicious for genetic disease, it is desirable to obtain molecular diagnosis before performing HSCT, since it can influence the transplant procedure. However, if HSCT has to be performed without delay for clinical indication, related donors should be excluded to avoid the risk of relapse or partial benefit due to a hereditary genetic defect.


Assuntos
Adenosina Desaminase/deficiência , Síndrome Linfoproliferativa Autoimune/terapia , Transplante de Células-Tronco Hematopoéticas , Peptídeos e Proteínas de Sinalização Intercelular/deficiência , Condicionamento Pré-Transplante , Doadores não Relacionados , Adenosina Desaminase/imunologia , Apoptose/efeitos dos fármacos , Apoptose/imunologia , Síndrome Linfoproliferativa Autoimune/enzimologia , Síndrome Linfoproliferativa Autoimune/imunologia , Síndrome Linfoproliferativa Autoimune/patologia , Pré-Escolar , Feminino , Fator Estimulador de Colônias de Granulócitos/administração & dosagem , Humanos , Peptídeos e Proteínas de Sinalização Intercelular/imunologia , Neutropenia/enzimologia , Neutropenia/imunologia , Neutropenia/patologia , Neutropenia/terapia , Transplante Homólogo , Receptor fas/imunologia
13.
Eur J Haematol ; 95(4): 308-15, 2015 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-25402872

RESUMO

OBJECTIVES: Shwachman-Diamond syndrome is a rare disorder characterized by exocrine pancreatic insufficiency, skeletal abnormalities, and bone marrow failure, with high risk of leukemic evolution. The aim of the study was the immunophenotypic characterization of bone marrow cells from patients with Shwachman-Diamond syndrome to assess the maturation pathway of blood progenitor cells and to identify the presence of recurrent abnormalities. METHODS: Bone marrow samples from nineteen patients and eleven controls were analyzed by multiparameter flow cytometry. RESULTS: We found a low frequency of CD34+ cells (P = 0.0179) and myeloid progenitors (P = 0.025), in the bone marrow of patients with Shwachman-Diamond syndrome as compared to the controls. A significant reduction in the percentage of granulocytes (P = 0.002) and an increase of monocytes (P < 0.001) were also evident in the bone marrow of patients. CONCLUSIONS: On the basis of these observations, future prospective assessments may be useful to verify the contribution of bone marrow immunophenotype in the early identification of the evolution toward aplasia or myelodysplasia.


Assuntos
Doenças da Medula Óssea/diagnóstico , Doenças da Medula Óssea/metabolismo , Insuficiência Pancreática Exócrina/diagnóstico , Insuficiência Pancreática Exócrina/metabolismo , Hematopoese , Imunofenotipagem , Lipomatose/diagnóstico , Lipomatose/metabolismo , Adolescente , Adulto , Antígenos CD/metabolismo , Medula Óssea/patologia , Células da Medula Óssea/metabolismo , Células da Medula Óssea/patologia , Doenças da Medula Óssea/genética , Estudos de Casos e Controles , Diferenciação Celular , Linhagem da Célula , Criança , Pré-Escolar , Insuficiência Pancreática Exócrina/genética , Feminino , Citometria de Fluxo , Hematopoese/genética , Células-Tronco Hematopoéticas/citologia , Células-Tronco Hematopoéticas/metabolismo , Humanos , Lactente , Cariótipo , Lipomatose/genética , Masculino , Mutação , Síndrome de Shwachman-Diamond , Adulto Jovem
14.
Int J Hematol ; 99(2): 208-12, 2014 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-24338706

RESUMO

A twin pair affected by juvenile myelomonocytic leukemia (JMML) with the same somatic PTPN11 mutation and abnormal chromosome 7 in bone marrow samples but distinct prognostic gene expression signatures, received a matched-unrelated donor and matched-unrelated cord blood transplant, respectively. Both twins fully engrafted, but after 6 months, the twin with an acute-myeloid-like (AML-like) signature at diagnosis rejected the graft and had an autologous reconstitution. A second transplant with an unrelated 5/6-HLA-matched-loci cord blood performed after 4 months from rejection was unsuccessful. After 25 months from diagnosis, the twin with the AML-like gene expression signature died of liver failure while on progression of his JMML. The other twin, who had a non-acute-myeloid-like (non-AML-like) gene expression signature at diagnosis is in complete hematological remission with full donor chimera. This observation suggests a biological diversity of JMML also in patients with a common genetic background.


Assuntos
Doenças em Gêmeos/terapia , Rejeição de Enxerto/fisiopatologia , Sobrevivência de Enxerto , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Leucemia Mielomonocítica Juvenil/terapia , Transplante de Células-Tronco de Sangue do Cordão Umbilical/efeitos adversos , Doenças em Gêmeos/diagnóstico , Doenças em Gêmeos/imunologia , Doenças em Gêmeos/metabolismo , Evolução Fatal , Perfilação da Expressão Gênica , Regulação Leucêmica da Expressão Gênica , Rejeição de Enxerto/imunologia , Humanos , Lactente , Leucemia Mielomonocítica Juvenil/diagnóstico , Leucemia Mielomonocítica Juvenil/imunologia , Leucemia Mielomonocítica Juvenil/metabolismo , Masculino , Prognóstico , Indução de Remissão , Transplante Homólogo , Resultado do Tratamento , Gêmeos Monozigóticos
15.
J Child Neurol ; 27(7): 867-74, 2012 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-22241706

RESUMO

The objective of this study was to evaluate whether electroneurography could help in differentiating between vincristine-induced neuropathy and acute inflammatory demyelinating polyradiculoneuropathy. We performed electroneurography in 7 children from September 2006 to March 2009 admitted to receive chemotherapy including vincristine for acute lymphoblastic leukemia, in whom severe acute limb weakness developed, suggesting vincristine-induced neuropathy. Three of 7 patients had electroneurography, suggesting acute inflammatory demyelinating polyradiculoneuropathy. They received intravenous immunoglobulins without discontinuing chemotherapy, and within 10 days their electroclinical conditions improved. Although electroneurography showed only absent F waves, preventing us from reaching a definitive neurophysiological diagnosis of acute inflammatory demyelinating polyradiculoneuropathy, children's presenting clinical manifestations, their disease course, and rapid and complete recovery after intravenous immunoglobulins argued strongly in its favor. A prompt, correct differential diagnosis of vincristine neuropathy and acute inflammatory demyelinating polyradiculoneuropathy in patients with acute lymphoblastic leukemia receiving vincristine is essential to improve disease outcome and prolong life expectancy.


Assuntos
Antineoplásicos Fitogênicos/efeitos adversos , Síndrome de Guillain-Barré/diagnóstico , Polineuropatias/induzido quimicamente , Polineuropatias/diagnóstico , Vincristina/efeitos adversos , Criança , Pré-Escolar , Estimulação Elétrica/métodos , Feminino , Lateralidade Funcional/efeitos dos fármacos , Síndrome de Guillain-Barré/tratamento farmacológico , Humanos , Imunoglobulinas/administração & dosagem , Masculino , Condução Nervosa/efeitos dos fármacos , Condução Nervosa/fisiologia , Nervo Fibular/fisiopatologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Tempo de Reação/efeitos dos fármacos , Estudos Retrospectivos , Fatores de Tempo
16.
Fundam Clin Pharmacol ; 17(1): 125-31, 2003 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-12588640

RESUMO

The off-label and unlicensed use of drugs to treat children is a common practice that occurs either in hospital or in the community. This derives from the fact that research for establishing drug efficacy and safety in children has not been carried out due to ethical problems, logistical difficulties, financial and legal concerns. In this work we report the studies available in literature documenting the extent of drug use in the paediatric field outside the recommendations of the license. From our analysis, a widespread attitude to prescribe medicines to children outside their product license either in the hospitals or in the community is confirmed. This suggests an immediate action for a more rationale use of drugs in paediatrics, to avoid exposing children and infants to unnecessary risks, but also to avoid depriving them of potentially effective and sometimes life-saving therapies.


Assuntos
Aprovação de Drogas , Rotulagem de Medicamentos , Revisão de Uso de Medicamentos , Criança , Tratamento Farmacológico , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos , Humanos , Padrões de Prática Médica
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