RESUMO
Osteonecrosis (ON) is a known complication of acute leukemia (AL) management, affecting 1%-10% of young patients and resulting in long-term morbidity. Widespread access to MRI over the past decade has allowed earlier detection and more accurate assessment. This study investigated clinical and MRI features of the 129 (2.5%) patients with symptomatic ON retrospectively recruited from the French LEA (Leucémies de l'Enfant et de l'Adolescent, or child and adolescent leukemias) cohort (n = 4,973). We analyzed data concerning ON risk factors, multifocal involvement, severe lesions detected by MRI, and patient quality of life (QoL). ON patients tended to be >10 years old at the time of AL diagnosis (odds ratio [OR]: 22.46; p < 10-6), female (OR: 1.8; p = 0.002), or treated for relapse (OR: 1.81; p = 0.041). They more frequently suffered from other sequelae (p < 10-6). Most necroses involved weight-bearing joints, and they were multifocal in 69% of cases. Double-blinded review of MRIs for 39 patients identified severe lesions in 14, usually in the hips. QoL of adolescents and adults was poor and permanently impacted after onset of ON. In conclusion, age >10 at time of AL diagnosis, female sex, and relapse occurrence were risk factors for multifocal ON; MRI revealed severe ON in a third of the patients considered; and ON was associated with persistently poor QoL affecting multiple domains. Future studies should include prospective data addressing ON management and seek to identify genetic markers for targeted screening enabling early ON detection and treatment.
Assuntos
Leucemia Mieloide Aguda , Osteonecrose , Criança , Adulto , Humanos , Adolescente , Feminino , Qualidade de Vida , Estudos Prospectivos , Estudos Retrospectivos , Seguimentos , Sobreviventes , Leucemia Mieloide Aguda/epidemiologia , Doença Aguda , Osteonecrose/diagnóstico por imagem , Osteonecrose/epidemiologia , Osteonecrose/etiologia , RecidivaRESUMO
BACKGROUND: This study aimed to evaluate the impact of patient's position on pain and anxiety during lumbar puncture (LP). MATERIALS AND METHODS: A randomized controlled trial included children between 2 and 18 years old receiving at least 2 therapeutic LPs. They were randomly assigned to undergo lateral decubitus position or sitting position LP. Primary outcome was the maximum LP-induced pain, secondary endpoint the maximum LP-induced anxiety score. RESULTS: Twenty-eight patients were randomized. For patients under 6 years old, mean of Face, Leg, Activity, Cry, and Consolability were 2.8/10±3.0 (median=1) at first time and 1.5±1.7 (median=1) at second time. For patients 6 to 18 years old, mean of visual analog scale were 2.2±2.2 (median=1.5) at first time and 3.2±2.8 (median=3) at second time. There was no significant differences according to position on anxiety among children. CONCLUSIONS: Results did not demonstrate whether lateral decubitus position could generate less pain and anxiety than sitting position.
Assuntos
Dor , Punção Espinal , Adolescente , Criança , Pré-Escolar , Humanos , Dor/etiologia , Medição da Dor , Projetos Piloto , Estudos Prospectivos , Punção Espinal/efeitos adversosRESUMO
We report survival and late effects analysis of TGM95 study for childhood (≤18 years) ovarian nonseminomatous germ cell tumors (NS-GCT). Patients with localized tumors (FIGO-stage IA) had no adjuvant treatment (low-risk, LR). Patients with advanced-stage received 3-5 VBP (vinblastin-bleomycin-cisplatin) in intermediate-risk group (IR: FIGO-stage IC-II-III and AFP < 15 000 ng/mL) or 4-6 VIP (etoposide-ifosfamide-cisplatin) in high-risk group (HiR: metastatic or AFP ≥ 15 000 ng/mL). Seventy-seven patients were included (median age = 12 years): 14 LR (13 FIGO-stage IA, 1 retrospectively IC), 26 IR (12 IC, 12 II-III, 2 not-available) and 37 HiR (2 IA with AFP ≥ 15 000 ng/mL, 27 II-III, 8 IV). After a median follow-up of 13.4 years, 12 events (eight relapses) and six deaths (two GCT-related, two due to acute myeloid leukemia and two noncancer related) occurred. All relapses (6 LR, 1 IR) occurred within 2 years. Four contralateral mature teratomas were observed within 8 years. Five-year EFS and OS were 88.2% (95%CI = 79-94%) and 94.6% (95%CI = 87-98%). Seven patients (9%) had bilateral gonadectomy. Among 51 survivors at 2 years aged >15 years (median = 26 years) with remaining ovarian tissue, all had developed spontaneous puberty and 21 (41%) had at least one pregnancy (including two with infertility treatment). Among 69 patients treated with platinum-based chemotherapy, chronic-kidney-disease was diagnosed in four patients (three after VIP) and significant ototoxicity occurred in three (all grade-2). Childhood ovarian NS-GCTs have an excellent prognosis with few late effects. The low-intensive etoposide-free VBP regimen could be an alternative in children with IR disease especially in cases of tumor rupture. The risk of contralateral mature teratoma needs regular monitoring of the remaining ovary.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Biomarcadores Tumorais/metabolismo , Neoplasias Embrionárias de Células Germinativas/terapia , Neoplasias Ovarianas/terapia , Ovariectomia/métodos , Neoplasias Testiculares/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Criança , Pré-Escolar , Gonadotropina Coriônica/metabolismo , Feminino , Seguimentos , França , Humanos , Neoplasias Embrionárias de Células Germinativas/metabolismo , Neoplasias Embrionárias de Células Germinativas/patologia , Neoplasias Ovarianas/metabolismo , Neoplasias Ovarianas/patologia , Prognóstico , Análise de Sobrevida , Neoplasias Testiculares/metabolismo , Neoplasias Testiculares/patologia , alfa-Fetoproteínas/metabolismoRESUMO
Outcomes in childhood T-cell acute lymphoblastic leukaemia (T-ALL) are steadily improving due to intensive therapy. Between 1989 and 2008, 599 children with newly diagnosed T-ALL were enrolled in two successive European Organization for Research and Treatment of Cancer - Children's Leukaemia Group trials (58881 and 58951), both based on the Berlin-Frankfurt-Munster protocol and without cranial irradiation. In the latter trial induction chemotherapy was intensified. The most important randomizations were Medac Escherichia coli asparaginase versus Erwinia asparaginase in trial 58881, and dexamethasone (6 mg/m2 /day) versus prednisolone (60 mg/m2 /day) and prolonged versus conventional asparaginase duration in trial 58951. 8-year event-free survival (EFS) increased from 65·1% to 74·0% in trial 58951. Improvement was most profound for patients with white blood cell (WBC) counts <100 × 109 /l and "good responders" to prephase. Medac E. coli asparaginase was associated with longer EFS [hazard ratio (HR) 0·54, P = 0·0015] and overall survival (HR 0·51, P = 0·0018). Induction therapy with dexamethasone did not improve EFS compared to prednisolone. Remarkably, intensification of central nervous system (CNS)-directed therapy in trial 58951 resulted in fewer bone marrow relapses, while the incidence of CNS relapses remained low. In summary, we showed that adequate asparaginase therapy, intensified induction treatment and intensification of CNS-directed chemotherapy can result in an improvement of outcome in T-ALL patients with good prephase response and initial WBC counts <100 × 109 /l, representing approximately 50% of T-ALL patients.
Assuntos
Ensaios Clínicos como Assunto/métodos , Leucemia-Linfoma Linfoblástico de Células T Precursoras/terapia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Leucemia-Linfoma Linfoblástico de Células T Precursoras/mortalidade , Análise de Sobrevida , Resultado do TratamentoRESUMO
The prevalence of the metabolic syndrome among adults from the French LEA childhood acute leukemia survivors' cohort was prospectively evaluated considering the type of anti-leukemic treatment received, and compared with that of controls. The metabolic profile of these patients was compared with that of controls. A total of 3203 patients from a French volunteer cohort were age- and sex-matched 3:1 to 1025 leukemia survivors (in both cohorts, mean age: 24.4 years; females: 51%). Metabolic syndrome was defined according to the National Cholesterol Education Program's Adult Treatment Panel III criteria. Metabolic syndrome was found in 10.3% of patients (mean follow-up duration: 16.3±0.2 years) and 4.5% of controls, (OR=2.49; P<0.001). Patients transplanted with total body irradiation presented the highest risk (OR=6.26; P<0.001); the other treatment groups also showed a higher risk than controls, including patients treated with chemotherapy only. Odd Ratios were 1.68 (P=0.005) after chemotherapy only, 2.32 (P=0.002) after chemotherapy and cranial irradiation, and 2.18 (P=0.057) in patients transplanted without irradiation. Total body irradiation recipients with metabolic syndrome displayed a unique profile compared with controls: smaller waist circumference (91 vs 99.6 cm; P=0.01), and increased triglyceride levels (3.99 vs 1.5 mmol/L; P<0.001), fasting glucose levels (6.2 vs 5.6 mmol/L; P=0.049), and systolic blood pressure (137.9 vs 132.8 mmHg; P=0.005). By contrast, cranial irradiation recipients with metabolic syndrome had a larger waist circumference (109 vs 99.6 cm; P=0.007) than controls. Regardless of the anti-leukemic treatment, metabolic syndrome risk was higher among childhood leukemia survivors. Its presentation differed depending on the treatment type, thus suggesting a divergent pathophysiology. This study is registered at clinicaltrials.gov identifier: 01756599.
Assuntos
Leucemia/complicações , Síndrome Metabólica/epidemiologia , Sobreviventes , Adulto , Antineoplásicos/uso terapêutico , Estudos de Casos e Controles , Irradiação Craniana , Feminino , França , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Masculino , Prevalência , Fatores de Risco , Triglicerídeos/sangue , Circunferência da Cintura , Irradiação Corporal Total , Adulto JovemRESUMO
In this retrospective study, we evaluate long-term complications in nearly all ß-thalassemia-major patients who successfully received allogeneic hematopoietic stem cell transplantation in France. Ninety-nine patients were analyzed with a median age of 5.9 years at transplantation. The median duration of clinical follow up was 12 years. All conditioning regimens were myeloablative, most were based on busulfan combined with cyclophosphamide, and more than 90% of patients underwent a transplant from a matched sibling donor. After transplantation, 11% of patients developed thyroid dysfunction, 5% diabetes, and 2% heart failure. Hypogonadism was present in 56% of females and 14% of males. Female patients who went on to normal puberty after transplant were significantly younger at transplantation than those who experienced delayed puberty (median age 2.5 vs 8.7 years). Fertility was preserved in 9 of 27 females aged 20 years or older and 2 other patients became pregnant following oocyte donation. In addition to patient's age and higher serum ferritin levels at transplantation, time elapsed since transplant was significantly associated with decreased height growth in multivariate analysis. Weight growth increased after transplantation particularly in females, 36% of adults being overweight at last evaluation. A comprehensive long-term monitoring, especially of endocrine late effects, is required after hematopoietic stem cell transplantation for thalassemia.
Assuntos
Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Talassemia beta/complicações , Talassemia beta/epidemiologia , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , França/epidemiologia , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Inquéritos Epidemiológicos , Transplante de Células-Tronco Hematopoéticas/métodos , Humanos , Masculino , Período Pós-Operatório , Prognóstico , Estudos Retrospectivos , Transplante Homólogo/efeitos adversos , Resultado do Tratamento , Adulto Jovem , Talassemia beta/terapiaRESUMO
To study the management of acute appendicitis in neutropenic patients, we retrospectively reviewed cases of acute appendicitis in neutropenic children treated for cancer. The patients' demographics, medical records, and outcomes were tracked. We compared nonoperative treatment versus emergency or delayed surgery. The cases of 30 patients with a mean age of 8.8 years in 12 French departments of Pediatric Hematology/Oncology between 1995 and 2013 were studied. Most patients (90%) were treated for hematological malignancies. Seven of the 30 children were successfully treated with exclusive medical treatment. Early surgery was performed in 6 patients, and the remaining 17 underwent combined management with a first-line antibiotic treatment and delayed appendectomy. Treatments were successful in all cases with transitory complications in only 3 patients. No death linked to infection was reported. Surgery was well tolerated even in the neutropenic period. Appendix perforation was a major risk factor of prolonged hospitalization. Histologic as well as bacteriologic and mycologic/parasitologic analyses were required in case of surgery. Analysis of diagnostic assessments showed the major importance of imaging (ultrasonography and tomography) on diagnosis confirmation. We could not come to a conclusion in the few numbers of reviewed cases because of a significant difference in management strategies, but we can conclude that early surgery after adequate supportive care is an acceptable modality of treatment and must be chosen in the face of life-threatening conditions.
Assuntos
Apendicite/complicações , Apendicite/terapia , Neutropenia/complicações , Adolescente , Apendicite/cirurgia , Criança , Pré-Escolar , Diagnóstico por Imagem , Feminino , Humanos , Lactente , Tempo de Internação , Masculino , Estudos Retrospectivos , Tempo para o Tratamento , Resultado do TratamentoRESUMO
Cardiovascular conditions are serious long-term complications of childhood acute leukemia. However, few studies have investigated the risk of metabolic syndrome, a known predictor of cardiovascular disease, in patients treated without hematopoietic stem cell transplantation. We describe the overall and age-specific prevalence, and the risk factors for metabolic syndrome and its components in the L.E.A. (Leucémie de l'Enfant et de l'Adolescent) French cohort of childhood acute leukemia survivors treated without hematopoietic stem cell transplantation. The study included 650 adult patients (mean age at evaluation: 24.2 years; mean follow-up after leukemia diagnosis: 16.0 years). The prevalence of metabolic syndrome was 6.9% (95% CI 5.1-9.2). The age-specific cumulative prevalence at 20, 25, 30 and 35 years of age was 1.3%, 6.1%, 10.8% and 22.4%, respectively. The prevalence of decreased high-density lipoprotein cholesterol, increased triglycerides, increased fasting glucose, increased blood pressure and increased abdominal circumference was 26.8%, 11.7%, 5.8%, 36.7% and 16.7%, respectively. Risk factors significantly associated with metabolic syndrome in the multivariate analysis were male sex (OR 2.64; 95% CI 1.32-5.29), age at last evaluation (OR 1.10; 95% CI 1.04-1.17) and body mass index at diagnosis (OR 1.15; 95% CI 1.01-1.32). The cumulative steroid dose was not a significant risk factor. Irradiated and non-irradiated patients exhibited different patterns of metabolic abnormalities, with more frequent abdominal obesity in irradiated patients and more frequent hypertension in non-irradiated patients. Survivors of childhood acute leukemia are at risk of metabolic syndrome, even when treated without hematopoietic stem cell transplantation or central nervous system irradiation. A preventive approach with regular screening for cardiovascular risk factors is recommended. clinicaltrials.gov identifier:01756599.
Assuntos
Leucemia/complicações , Síndrome Metabólica/epidemiologia , Síndrome Metabólica/etiologia , Sobreviventes , Adolescente , Adulto , Fatores Etários , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , Estudos de Coortes , Terapia Combinada , Feminino , Seguimentos , Humanos , Leucemia/terapia , Masculino , Prevalência , Radioterapia/efeitos adversos , Radioterapia/métodos , Indução de Remissão , Fatores de Risco , Adulto JovemRESUMO
Relapse after transplantation is a major cause of treatment failure in paediatric acute lymphoblastic leukaemia (ALL). Here, we report the findings of a prospective national study designed to investigate the feasibility of immune intervention in children in first or subsequent remission following myeloablative conditioning. This study included 133 children who received a transplant for ALL between 2005 and 2008. Minimal Residual Disease (MRD) based on T cell receptor/immunoglobulin gene rearrangements was measured on days -30, 30, 90 and 150 post-transplantation. Ciclosporin treatment was rapidly discontinued and donor lymphocyte infusions (DLI) were programmed for patients with a pre- or post-transplant MRD status ≥10(-3) . Only nine patients received DLI. Pre- and post-transplant MRD status, and the duration of ciclosporin were independently associated with 5-year overall survival (OS), which was 62·07% for the whole cohort. OS was substantially higher in patients cleared of MRD than in those with persistent MRD (52·3% vs. 14·3%, respectively). Only pre-transplant MRD status (Hazard Ratio 2·57, P = 0·04) and duration of ciclosporin treatment (P < 0·001) were independently associated with relapse. The kinetics of chimerism were not useful for predicting relapse, whereas MRD monitoring up to 90 d post-transplantation was a valuable prognostic tool to guide therapeutic intervention.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Quimeras de Transplante , Transferência Adotiva , Criança , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Linfócitos , Masculino , Neoplasia Residual , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Prognóstico , Modelos de Riscos Proporcionais , Doadores de Tecidos , Resultado do TratamentoRESUMO
DNA copy number analysis has been instrumental for the identification of genetic alterations in B-cell precursor acute lymphoblastic leukemia. Notably, some of these genetic defects have been associated with poor treatment outcome and might be relevant for future risk stratification. In this study, we characterized recurrent deletions of CD200 and BTLA genes, mediated by recombination-activating genes, and used breakpoint-specific polymerase chain reaction assay to screen a cohort of 1154 cases of B-cell precursor acute lymphoblastic leukemia uniformly treated according to the EORTC-CLG 58951 protocol. CD200/BTLA deletions were identified in 56 of the patients (4.8%) and were associated with an inferior 8-year event free survival in this treatment protocol [70.2% ± 1.2% for patients with deletions versus 83.5% ± 6.4% for non-deleted cases (hazard ratio 2.02; 95% confidence interval 1.23-3.32; P=0.005)]. Genetically, CD200/BTLA deletions were strongly associated with ETV6-RUNX1-positive leukemias (P<0.0001), but were also identified in patients who did not have any genetic abnormality that is currently used for risk stratification. Within the latter population of patients, the presence of CD200/BTLA deletions was associated with inferior event-free survival and overall survival. Moreover, the multivariate Cox model indicated that these deletions had independent prognostic impact on event-free survival when adjusting for conventional risk criteria. All together, these findings further underscore the rationale for copy number profiling as an important tool for risk stratification in human B-cell precursor acute lymphoblastic leukemia. This trial was registered at www.ClinicalTrials.gov as #NCT00003728.
Assuntos
Antígenos CD/genética , Deleção de Genes , Leucemia-Linfoma Linfoblástico de Células Precursoras B/genética , Receptores Imunológicos/genética , Adolescente , Alelos , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , Pontos de Quebra do Cromossomo , Ensaios Clínicos como Assunto , Hibridização Genômica Comparativa , Variações do Número de Cópias de DNA , Feminino , Frequência do Gene , Humanos , Lactente , Recém-Nascido , Estimativa de Kaplan-Meier , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras B/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras B/mortalidade , Prognóstico , RecidivaRESUMO
We evaluated the impact of central nervous system irradiation (CNSI) on long-term health status and quality of life (QoL) of childhood lymphoblastic leukemia survivors included in the French L.E.A. (Childhood and Adolescent Leukemia) multicentric cohort. QoL was self-reported in adults and assessed by parents in children and adolescents, using adapted questionnaires. From 2004 to 2009, 630 nongrafted patients were assessed after 11.8±6.3 years from diagnosis. Patients receiving CNSI (18.6%) or chemotherapy alone (81.4%) were compared. The risk of having long-term physical effects was increased with CNSI (odds ratio=3.3; 95% confidence interval, 1.8-5.9), especially regarding growth failure, second tumor, cataract, and overweight. QoL did not differ significantly according to the treatment received, despite a tendency toward lower scores with CNSI in children and adolescents (summary score 63.6±13.3 vs. 71.7±12.4, P=0.14). Compared with French norms, adult survivors had an impaired QoL, especially in mental domains (mental composite score 45.2±9.8 vs. 47.9±2.1, P<0.001). In pediatric survivors, QoL was not impaired and even tended to be higher than population norms (summary score 71.7±12.4 vs. 70.0±4.2, P=0.054), mainly in social and relational domains. In conclusion, QoL seems to be impaired by the trauma of a life-threatening illness in childhood, as well as by the treatment received.
Assuntos
Neoplasias Encefálicas/psicologia , Irradiação Craniana/efeitos adversos , Leucemia-Linfoma Linfoblástico de Células Precursoras/psicologia , Qualidade de Vida , Sobreviventes/psicologia , Doença Aguda , Adolescente , Adulto , Neoplasias Encefálicas/etiologia , Neoplasias Encefálicas/mortalidade , Criança , Pré-Escolar , Feminino , Seguimentos , Nível de Saúde , Humanos , Lactente , Recém-Nascido , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Leucemia-Linfoma Linfoblástico de Células Precursoras/radioterapia , Prognóstico , Estudos Prospectivos , Inquéritos e Questionários , Taxa de Sobrevida , Tempo , Adulto JovemRESUMO
Minimal residual disease (MRD) is a major predictive factor of the cure rate of acute lymphoblastic leukaemia (ALL). Haematopoietic cell transplantation is a treatment option for patients at high risk of relapse. Between 2005 and 2008, we conducted a prospective study evaluating the feasibility and efficacy of the reduction of immunosuppressive medication shortly after a non-ex vivo T depleted myeloablative transplantation. Immunoglobulin (Ig)H/T-cell receptor MRD 30 d before transplant could be obtained in 122 of the 133 cases of high-risk paediatric ALL enrolled. There were no significant demographic differences except remission status (first or second complete remission) between the 95 children with MRD <10(-3) and the 27 with MRD ≥10(-3) . Multivariate analysis identified sex match and MRD as being significantly associated with 5-year survival. MRD ≥10(-3) compromised the 5-year cumulative incidence of relapse (43·6 vs. 16·7%). Complete remission status and stem cell source did not modify the relationship between MRD and prognosis. Thus, pre-transplant MRD is still a major predictor of outcome for ALL. The MRD-guided strategy resulted in survival for 72·3% of patients with MRD<10(-3) and 40·4% of those with MRD ≥10(-3).
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas/métodos , Neoplasia Residual/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adjuvantes Imunológicos/administração & dosagem , Adolescente , Criança , Pré-Escolar , Terapia Combinada , Intervalo Livre de Doença , Feminino , Humanos , Masculino , Neoplasia Residual/imunologia , Neoplasia Residual/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/imunologia , Prognóstico , Estudos Prospectivos , Indução de Remissão , Resultado do TratamentoRESUMO
Neonatal acute myeloid leukemias (AML) occurred within the first 28 days of life and constitute only a small proportion of all AL. They are distinguished from leukemias of older children by their clinical presentation, which frequently includes cutaneous localizations ("blueberry muffin rash syndrome") and a leukocytosis above 50 ×109/L. This proliferation may be transient, causing a transient leukemoid reaction in a background of constitutional trisomy 21 ("Transient Abnormal Myelopoieseis" or TAM) or Infantile Myeloproliferative Disease in the absence of constitutional trisomy 21 ("Infantile Myeloproliferative Disease" or IMD). In cases of true neonatal AML, the prognosis of patients is poor. Overall survival is around 35 % in the largest historical series. This poor prognosis is mainly due to the period of onset of this pathology making the use of chemotherapy more limited and involving many considerations, both ethical and therapeutic. The objective of this work is to review this rare pathology by addressing the clinical, biological, therapeutic and ethical particularities of patients with true neonatal AML or transient leukemoid reactions occurring in a constitutional trisomy 21 (true TAM) or somatic background (IMD).
Assuntos
Síndrome de Down , Leucemia Mieloide Aguda , Humanos , Leucemia Mieloide Aguda/terapia , Leucemia Mieloide Aguda/diagnóstico , Recém-Nascido , Síndrome de Down/terapia , Prognóstico , Reação Leucemoide/terapia , Reação Leucemoide/diagnóstico , Transtornos Mieloproliferativos/terapia , Transtornos Mieloproliferativos/diagnóstico , Transtornos Mieloproliferativos/genéticaRESUMO
Chemotherapy-induced nausea and vomiting (CINV) are frequent and dreaded side effects in cancer treatments. CINV has a major impact on patient's condition and quality of life. Prophylaxis is tailored to patient's profile and the emetogenic level of their chemotherapy. The aim of this study is to update the recommendations for CINV prevention and management in pediatric onco-hematology for use in France, by adapting the guidelines of the Pediatric Oncology Group of Ontario (POGO). Clinical practice guideline adaptation is a recognized method for tailoring existing clinical practice guidelines to local context. A multidisciplinary French-speaking panel was formed to discuss about POGO guideline recommendations for the acute and delayed phases, breakthrough, refractory and anticipatory CINV and the evidence supporting them. Panel members were asked whether they wanted to adopt, modify or reject each of the POGO guideline recommendations. Panel members translated each recommendation and adapted recommendations for an implementation in France. Their acceptance required agreement at least 80 % of panel members. Algorithms and tables were created, listing all the recommendations and providing a better overview for decision-making process adapted to the patient's profile. These recommendations should be reviewed for implementation at French institutions caring for pediatric cancer patients and once implemented, the rates of adherence to recommendations and CINV control should be reported.
Assuntos
Antieméticos , Antineoplásicos , Náusea , Neoplasias , Vômito , Humanos , Vômito/induzido quimicamente , Vômito/prevenção & controle , Náusea/induzido quimicamente , Náusea/prevenção & controle , Criança , Antineoplásicos/efeitos adversos , Adolescente , Neoplasias/tratamento farmacológico , França , Antieméticos/uso terapêutico , Algoritmos , Sociedades MédicasRESUMO
The late effects and quality of life (QoL) in childhood acute leukemia survivors were compared between hematopoietic stem cell transplantation (HSCT) recipients and patients who underwent conventional therapy. The study included 943 patients, 256 of whom underwent HSCT (27.1%). Medical visits were conducted to detect the occurrence of physical late effects. Based on patient age, different questionnaires were used to assess QoL. To evaluate the association between HSCT and each type of late effect or QoL dimension, the appropriate multivariate regressions were performed. QoL mean scores were compared with those obtained for age- and sex-matched French control subjects. Of all the survivors, 674 (71.5%) had at least 1 late effect, with the risk being 5.0 CI95 (3.0-8.6) times higher for transplantation survivors. For child survivors, scoring of QoL showed no significant differences between the treatment groups. The adult HSCT survivors reported lower physical dimension QoL scores than chemotherapy survivors. Compared with French norms, the survivor group reported a significantly lower mental composite score; however, the physical composite score showed no significant difference. Thus, transplanted survivors have a high risk of developing late effects, resulting in a decreased physical well-being in adulthood. However, long after treatment completion, childhood leukemia survivors report that effects on psychological well-being are more important than they are in physical QoL dimensions.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras/psicologia , Sobreviventes/psicologia , Adolescente , Antineoplásicos/uso terapêutico , Criança , Feminino , França , Humanos , Masculino , Análise Multivariada , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Qualidade de Vida , Projetos de Pesquisa , Estudos Retrospectivos , Inquéritos e Questionários , Transplante HomólogoRESUMO
Femoral and lumbar bone mineral densities (BMDs) were measured in 159 adults enrolled in the Leucémies de l'Enfant et de l'Adolescent program, a French prospective multicentric cohort of childhood leukemia survivors. BMDs were expressed as Z-scores, and multivariate linear regression analyses were used to construct association models with potential risk factors. Mean age at evaluation and follow-up was 23 and 14.7 years, respectively. In the whole cohort, mean femoral Z-score was -0.19 ± 0.08. Two factors were associated with lower femoral BMD transplantation (-0.49 ± 0.15 vs -0.04 ± 0.10 in the chemotherapy group; P = .006) and female sex (-0.34 ± 0.10 vs -0.03 ± 0.13; P = .03). Among patients who received a transplant, the only significant risk factor was hypogonadism (-0.88 ± 0.16 vs -0.10 ± 0.23; P = .04). A slight reduction in lumbar BMD (mean Z-score, -0.37 ± 0.08) was detected in the whole cohort without difference between the transplantation and chemotherapy groups. Among patients who received a transplant, younger age at transplantation was correlated with a low lumbar BMD (P = .03). We conclude that adults who had received only chemotherapy for childhood leukemia have a slight reduction in their lumbar BMD and a normal femoral BMD. Patients who received a transplant with gonadal deficiency have a reduced femoral BMD which might increase the fracture risk later in life.
Assuntos
Densidade Óssea , Leucemia Mieloide/metabolismo , Leucemia-Linfoma Linfoblástico de Células Precursoras/metabolismo , Sobreviventes , Absorciometria de Fóton , Doença Aguda , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Dexametasona/administração & dosagem , Dexametasona/efeitos adversos , Feminino , Seguimentos , Transplante de Células-Tronco Hematopoéticas , Humanos , Leucemia Mieloide/terapia , Modelos Lineares , Masculino , Análise Multivariada , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Prednisona/administração & dosagem , Prednisona/efeitos adversos , Estudos Prospectivos , Adulto JovemRESUMO
Organization of health care of a patient followed in pediatric oncology is not limited to cancer treatment. It includes a whole range of supportive care. Some are common to the supportive care offered in adult oncology, such as pain management and nutritional support. However, there are pediatric specificities. Others are more peculiar to children, such as education and information for young patients, and require a specific framework and innovative tools. The young age of patients and the improvement in survival rates in pediatric oncology also lead to questioning the temporality of supportive care by considering access to supportive care beyond the active phase of the disease. This review explains some of these different specificities: information and communication to the patient and his parents, assessment and management of pain, nutritional support but also schooling, long-term follow-up and screening sequelae induced by the disease and its treatments.
Assuntos
Oncologia , Neoplasias , Adulto , Criança , Comunicação , Humanos , Neoplasias/complicações , Neoplasias/diagnóstico , Neoplasias/terapia , Manejo da Dor , PaisRESUMO
Pegaspargase (Oncaspar®), a pegylated form of native Escherichia Coli-derived L-asparaginase is an essential component chemotherapy used in the treatment of acute lymphoblastic leukemia (ALL) in pediatric and adult patients. Its particular toxicity profile requires a specific management to improve safety and tolerability and optimize treatment outcome and therefore survival. Within the framework of workshops of practice harmonization of the French Society of Children and Adolescent Cancers, diagnostic and management of the most commonly occuring toxicities (excluding coagulation abnormalities) during Pegaspargase treatment were reviewed according to the analysis of published studies.
Assuntos
Antineoplásicos , Transtornos da Coagulação Sanguínea , Leucemia-Linfoma Linfoblástico de Células Precursoras , Adulto , Humanos , Adolescente , Criança , Asparaginase/efeitos adversos , Polietilenoglicóis/efeitos adversos , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Resultado do Tratamento , Antineoplásicos/efeitos adversosRESUMO
Nausea and vomiting induced by cancer treatment (CINV) remain one of the most common and feared side effects in children despite the use of new drugs to prevent them. The existing recommendations for the prophylaxis and treatment of CINV are based on adult patients in Anglo-Saxon societies. Based on a recent review of the literature, we focused on specific pediatric issues in order to offer recommendations validated by the supportive care committee of the French society for childhood cancer.
Assuntos
Antieméticos , Antineoplásicos , Hematologia , Neoplasias , Adulto , Criança , Humanos , Antieméticos/efeitos adversos , Neoplasias/tratamento farmacológico , Náusea/induzido quimicamente , Náusea/prevenção & controle , Vômito/induzido quimicamente , Vômito/prevenção & controle , Antineoplásicos/efeitos adversosRESUMO
Ten to fifteen percent of children with acute lymphoblastic leukemia (ALL) relapse following treatment. Of these, less than 2% display ophthalmic relapses, which owing to their scarcity, are largely undocumented, leaving clinicians with few diagnostic and therapeutic recommendations, despite serious functional sequelae. We conducted a French multicenter retrospective study to collect all clinical, radiological, biological, and therapeutic data, and outcomes for children with ALL ophthalmic relapses. From 2000 to 2020, 20 ophthalmic relapses occurring after first-line therapy performed before January 1st, 2017 were included in our study: 14 B-ALL and 6 T-ALL. Fifteen patients (75%) had concomitant involvement of the central nervous system, and 11 (55%) a combined bone marrow relapse. Only 1 had an isolated ophthalmic relapse. Eight children (40%) died, 7 from a refractory disease and 1 from toxic death, and 4 patients relapsed. With a median follow-up of 63.1 months, 8 patients are currently alive in continuous complete remission with only 2 displaying severe ophthalmic sequelae. Although rare, ophthalmic relapse could have a significant impact on the functional prognosis of survivors. Their management must be multidisciplinary, with a central role given to ophthalmologists.