RESUMEN
As almost all patients with high-risk neuroblastomas have autograft, we aimed to determine if minimal residual disease (MRD) quantified by RT-PCR for tyrosine hydroxylase in PBSC is prognostic in neuroblastomas. PBSC harvests from 38 children were analyzed. Seven had harvests positive for TH-mRNA. Patients with a positive MRD had a lower 2-year-overall-survival compared to those with negative MRD (P = 0.04) regardless of whether or not PBSC were re-infused. Patients in CR/VGPR group with positive MRD have hazard ratio of death at 7.3 [1.3-40.5]. In conclusion, molecular MRD status in PBSC of good response group may be of interest as a survival prognostic factor in high-risk neuroblastomas.
Asunto(s)
Neuroblastoma/mortalidad , Neuroblastoma/terapia , Trasplante de Células Madre de Sangre Periférica , Niño , Preescolar , Supervivencia sin Enfermedad , Femenino , Humanos , Lactante , Masculino , Proteínas de Neoplasias/genética , Proteínas de Neoplasias/metabolismo , Neoplasia Residual , Neuroblastoma/enzimología , Neuroblastoma/genética , Estudios Prospectivos , ARN Mensajero/genética , ARN Mensajero/metabolismo , ARN Neoplásico/genética , ARN Neoplásico/metabolismo , Tasa de Supervivencia , Trasplante Autólogo , Tirosina 3-Monooxigenasa/genética , Tirosina 3-Monooxigenasa/metabolismoRESUMEN
Clinical grading of GI involvement during acute GVHD remains a challenging issue, especially in children. Plasma citrulline, a non-protein amino acid selectively produced and released by enterocytes, is a suitable surrogate endpoint for small intestinal epithelial cell mass, irrespective of the underlying cause of cell loss. Children referred for allogeneic bone marrow transplantation who were free from chronic malabsorption or constitutional disease involving the GI tract were consecutively included in this prospective study. Plasma citrulline and albumin concentration was measured every week between day 7 and day 28 of BMT until resolution of the aGVHD or occurrence of chronic GVHD. In total, 31 children were included between 2008 and 2011. After a CR, citrulline levels fell to a minimum level on day 7 and then increased to reach the initial value on day 28. After day 28, plasma citrulline but not albumin was strongly linked to the occurrence of GI GVHD, the threshold being set at 10 µmol/L. The correlation with clinical grade of GI-aGVHD now needs to be assessed in larger populations. In pediatric patients, citrulline is valuable as a suitable non-invasive marker of GI involvement in acute GVHD.
Asunto(s)
Trasplante de Médula Ósea/efectos adversos , Citrulina/sangre , Enfermedad Injerto contra Huésped/sangre , Enfermedad Injerto contra Huésped/diagnóstico , Adolescente , Albúminas/química , Biomarcadores/metabolismo , Niño , Preescolar , Diarrea/diagnóstico , Femenino , Humanos , Lactante , Masculino , Estudios Prospectivos , Factores de Tiempo , Resultado del TratamientoRESUMEN
Little information is available on erythrocytapheresis in children with sickle cell disease, and no comparison has ever been made with manual exchanges in a long-term blood exchange program. We matched a historical cohort of five patients who received 60 erythrocytapheresis procedures with five who received 124 manual exchanges. Long-term erythrocytapheresis was feasible and well-tolerated even in children of low weight. In a long-term approach, automated exchanges were more efficient in maintaining a low HbS level, and exchanges could be spaced out. This approach appears especially useful in the cases where the HbS level must be maintained below 30%.
Asunto(s)
Anemia de Células Falciformes/terapia , Transfusión de Eritrocitos , Adolescente , Anemia de Células Falciformes/sangre , Niño , Preescolar , Femenino , Hemoglobina Falciforme/metabolismo , Humanos , Masculino , Estudios Retrospectivos , Factores de TiempoRESUMEN
BACKGROUND: Outcomes of Ewing tumor (ET) patients treated with allogeneic stem cell transplantation (allo-SCT) were compared regarding the use of reduced-intensity conditioning (RIC) and high-intensity conditioning (HIC) regimens as well as human leukocyte antigen (HLA)-matched and HLA-mismatched grafts. PATIENTS AND METHODS: We retrospectively analyzed data of 87 ET patients from the European Group for Blood and Marrow Transplantation, Pediatric Registry for Stem Cell Transplantations, Asia Pacific Blood and Marrow Transplantation and MetaEICESS registries treated with allo-SCT. Fifty patients received RIC (group A) and 37 patients received HIC (group B). Twenty-four patients received HLA-mismatched grafts and 63 received HLA-matched grafts. RESULTS: Median overall survival was 7.9 months [±1.24, 95% confidence interval (CI) 5.44-10.31] for group A and 4.4 months (±1.06, 95% CI 2.29-6.43) for group B patients (P = 1.3). Death of complications (DOC) occurred in 4 of 50 (0.08) and death of disease (DOD) in 33 of 50 (0.66) group A and in 16 of 37 (0.43) and 17 of 37 (0.46) group B patients, respectively. DOC incidence was decreased (P < 0.01) and DOD/relapse increased (P < 0.01) in group A compared with group B. HLA mismatch was not generally associated with graft-versus-Ewing tumor effect (GvETE). CONCLUSIONS: There was no improvement of survival with RIC compared with HIC due to increased DOD/relapse incidence after RIC despite less DOC incidence. This implicates general absence of a clinically relevant GvETE with current protocols.
Asunto(s)
Neoplasias Óseas/mortalidad , Neoplasias Óseas/terapia , Enfermedad Injerto contra Huésped/terapia , Sarcoma de Ewing/mortalidad , Sarcoma de Ewing/terapia , Trasplante de Células Madre , Adolescente , Adulto , Niño , Preescolar , Femenino , Estudios de Seguimiento , Humanos , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Tasa de Supervivencia , Acondicionamiento Pretrasplante , Trasplante Homólogo , Resultado del Tratamiento , Adulto JovenRESUMEN
We report results of RIC AHSCT in four adolescents with aggressive refractory HL. They all received three or four lines of therapy prior to RIC-AHSCT including autografts. At the time of RIC, they were in partial response except for one patient who had progressive chemoresistant disease. The conditioning regimen consisted of fludarabin, busulfan and ATG. They all had a matched related donor. The median follow-up was 12-16-month post-allograft. All patient transplants engrafted rapidly. The median time of hospitalization was 35 days. The median time to neutrophil recovery (>or=500/muL) was 19 days. All the patients were in complete donor chimerism at day 60. Four patients developed skin (grade Asunto(s)
Busulfano/uso terapéutico
, Trasplante de Células Madre Hematopoyéticas/métodos
, Enfermedad de Hodgkin/cirugía
, Agonistas Mieloablativos/uso terapéutico
, Vidarabina/análogos & derivados
, Adolescente
, Antineoplásicos/uso terapéutico
, Quimioterapia Combinada
, Femenino
, Estudios de Seguimiento
, Enfermedad de Hodgkin/diagnóstico
, Enfermedad de Hodgkin/tratamiento farmacológico
, Humanos
, Masculino
, Tomografía de Emisión de Positrones
, Índice de Severidad de la Enfermedad
, Factores de Tiempo
, Trasplante Homólogo
, Resultado del Tratamiento
, Vidarabina/uso terapéutico
RESUMEN
To date, little is known about the fertility of women suffering from mucopolysaccharidosis type I (MPS I). We report on a female patient with MPS I treated by allogeneic bone marrow transplantation (BMT) at the age of 4 years (after a conditioning regimen containing busulfan 16 mg/kg and cyclophosphamide 100 mg/kg) who had four successful pregnancies without any reproductive assistance. Clinical and biological examinations of the children were normal. On the basis of this case, we discuss the fertility counselling of female MPS I patients at the time of BMT.
Asunto(s)
Trasplante de Médula Ósea , Mucopolisacaridosis I/complicaciones , Mucopolisacaridosis I/terapia , Complicaciones del Embarazo , Adolescente , Adulto , Preescolar , Femenino , Asesoramiento Genético , Humanos , Recién Nacido , Mucopolisacaridosis I/genética , Embarazo , Complicaciones del Embarazo/genética , Resultado del Embarazo , Adulto JovenRESUMEN
The objective of the study was to assess acute neurotoxicity associated with triple intrathecal therapy (TIT)+/-high-dose methotrexate (HD MTX) in children with acute lymphoblastic leukemia (ALL). 1395 children were enrolled on FRALLE 93 protocol from 1993 to 1999. Lower-risk group (LR, n=182) were randomized to weekly low-dose MTX at 25 mg/m(2)/week (LD MTX, n=81) or HD MTX at 1.5 g/m(2)/2 weeks x 6 (n=77). Intermediate-risk group (IR, n=672) were randomized to LD MTX (n=290) or HD MTX at 8 g/m(2)/2 weeks x 4 (n=316). Higher-risk group (HR, n=541) prednisone-responder patients received LD MTX and cranial radiotherapy. HR group steroid resistant cases were grafted (autologous or allogenic). TIT (MTX, cytarabine and methylprednisolone) was given every 2 weeks during 16-18 weeks and every 3 months during maintenance therapy in LR and IR patients. 52 patients (3.7%) developed neurotoxicity. Isolated seizures: n=15 (1.1%), peripheral and spinal neuropathy: n=17 (1.2%) and encephalopathy: n=20 (1.4%). Age >10 years was significantly associated with neurotoxicity (P=0.01) and use of HD MTX is associated with encephalopathy (P=0.03). Sequels are reported respectively in 60 and 33% of spinal neuropathy and encephalopathy cases. Current strategies tailoring risk of neurological sequels has to be defined.
Asunto(s)
Antimetabolitos Antineoplásicos/efectos adversos , Encefalopatías Metabólicas/inducido químicamente , Metotrexato/efectos adversos , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Adolescente , Factores de Edad , Encefalopatías Metabólicas/epidemiología , Encefalopatías Metabólicas/prevención & control , Niño , Preescolar , Terapia Combinada , Relación Dosis-Respuesta a Droga , Femenino , Humanos , Lactante , Masculino , Neurotoxinas , Leucemia-Linfoma Linfoblástico de Células Precursoras/radioterapia , Medición de RiesgoRESUMEN
In 10 years, the neonatal autoimmune enteropathy has been individualized from other causes of neonatal severe protracted diarrheas as a syndrome called Ipex for Immunodysregulation, Polyendocrinopathy, Enteropathy, X-linked. Thanks to linkage analyses in affected families, this rare paediatric syndrome with fatal outcome has been correlated to mutations of the foxp3 gene. Homozygous loss of function of foxp3 gene results in the absence of development of a crucial subpopulation of lymphocytes with CD4+CD25+ phenotype, called regulatory T-cells. The study of these lymphocytes allows a better understanding of the immune system homeostasis and of the physiopathology of Ipex syndrome, which is a prerequisite for treatment. Achieving ex vivo manipulation of such lymphocytes will end up on promising applications of cell therapy.
Asunto(s)
Enfermedades Autoinmunes/congénito , Enfermedades Autoinmunes/genética , Factores de Transcripción Forkhead/genética , Antígenos CD/inmunología , Enfermedades Autoinmunes/inmunología , Cromosomas Humanos X , Humanos , Recién Nacido , Linfocitos T Reguladores/inmunologíaRESUMEN
OBJECTIVE: Our objective was to improve the organization and management of care facilities for children suffering from cancer or leukaemia and to be aligned with the legislation in force in France. METHODS: Our report is on the successive steps for the implementation of a quality assurance system, methods used, motivations, cost, difficulties encountered as well as the advantages obtained. In the Regional Centre for Paediatric Oncology (CRCP) at the CHU in Clermont-Ferrand, we launched a quality programme based on ISO9001/2000 standards. The implementation of the quality assurance system was conducted as a research project and an established medical project with the support of the Management Team. The mission was divided into several "processes", an approach consisting of considering the clinical service in terms of flow and successions of transformations (reception, care, support, accompaniment, etc.) which produce added-value (services and products adapted to the needs of the "customers": children, families, correspondents). RESULTS: We singled out ten physical processes or "job specializations" such as "diagnosis", "care" or "project for the child". The cartography which is the systematic representation of the processes and the interactions between them made it possible to draw up a global vision of the CRCP "care" activity. CONCLUSION: The ISO9001/2000 standard is a tool designed to help organization and management. The benefit obtained in implementing it in a clinic was perceived in organisational terms and lead to a true team spirit, a standardization of the professional practices and the enhancement of the role of each person. The advantages appear at three levels: the child and his/her family, the medical and paramedical teams, and the administrative supervisory bodies.
Asunto(s)
Instituciones Oncológicas/normas , Oncología Médica/normas , Pediatría/normas , Garantía de la Calidad de Atención de Salud/organización & administración , Adolescente , Niño , Servicios de Salud del Niño/normas , Preescolar , Familia , Francia , Hospitales Universitarios , Humanos , Lactante , Recién Nacido , Auditoría Médica , Relaciones Profesional-Familia , Garantía de la Calidad de Atención de Salud/legislación & jurisprudencia , Garantía de la Calidad de Atención de Salud/normasRESUMEN
BACKGROUND: The immunomodulatory effect of extracorporeal photochemotherapy (photopheresis) coupled with its efficacy in lymphocytic skin diseases provides a rationale for its use for erosive lichen planus. We report two cases of chronic oral erosive and corticoresistant lichen planus successfully treated with photopheresis. PATIENTS AND METHODS: Case 1. A 61-years-old man had erosive oral lichen planus for four years. Oral steroids were contra-indicated due to iatrogenic pancreatitis. After nine photopheresis sessions, subjective improvement occurred and the oral lesions were stabilized. Case 2. A 17-years-old woman presented corticodependent oral and genital erosive lichen planus and cutaneous lesions. After seven sessions of photopheresis, the patient was able to eat again and the pain decreased. After 20 sessions, the cutaneous lichen planus disappeared and complete remission of the mucous lesions was obtained without corticotherapy. DISCUSSION: These two cases, together with 23 other cases reported elsewhere, strongly suggested the value of photopheresis in the treatment of erosive lichen planus. However, relapses after treatment withdrawal appear extremely frequent.
Asunto(s)
Corticoesteroides , Liquen Plano Oral/tratamiento farmacológico , Adolescente , Contraindicaciones , Femenino , Humanos , Masculino , Persona de Mediana Edad , Mucosa Bucal/efectos de los fármacos , Mucosa Bucal/patología , Pancreatitis/complicaciones , FotoféresisRESUMEN
Juvenile granulosa cell tumor (JGCT) in children accounted for 12% of all ovarian tumors treated in the Institut Gustave-Roussy (IGR) Pediatric Department from 1967 to 1985. The median age of the 15 girls was 8 years 7 months (range, 22 months to 15 years 7 months). Precocious pseudopuberty was present in six of the seven girls under 8 years. Of the other seven girls, one developed virilization symptoms. Surgery was the first treatment in each case. According to the Wollner classification, there were six stage I, one stage II, six stage III (including four ruptured tumors), and one stage IV JGCT cases. One patient was not available for staging. An adjuvant treatment (five chemotherapy and one radiotherapy combined with chemotherapy) was administered to six patients. Eleven girls are alive and free of disease, with a median follow-up of 6 years (range, 2 to 18 years). Four girls relapsed 6 to 17 months after surgery and died. Two of these relapses occurred in bone. The prognosis for JGCT in children is favorable for the lower stages when treated with surgery, but the best treatment for extensive and recurrent disease has yet to be determined.
Asunto(s)
Tumor de Células de la Granulosa/terapia , Neoplasias Ováricas/terapia , Adolescente , Niño , Preescolar , Terapia Combinada , Femenino , Tumor de Células de la Granulosa/mortalidad , Humanos , Lactante , Recurrencia Local de Neoplasia , Estadificación de Neoplasias , Neoplasias Ováricas/mortalidad , PronósticoRESUMEN
Twenty-one patients with advanced Wilms' tumor entered a phase II study with high-dose ifosfamide (3 g/m2 over two days every 15 days). Mesna and hyperhydration were associated with minimal bladder toxicity. After two courses, five partial responses and six complete responses were observed. Ten patients did not respond. The median duration of response was 2 months (range, 1 to 7). Therapy was delayed because of leukopenia for 1 or 2 weeks in only three cases. Fever and infection were not observed. Seven patients presented with hematuria, three of whom were among the 17 patients coadministered mesna, which did not interfere with subsequent therapy.
Asunto(s)
Ifosfamida/uso terapéutico , Neoplasias Renales/tratamiento farmacológico , Tumor de Wilms/tratamiento farmacológico , Adolescente , Adulto , Niño , Preescolar , Evaluación de Medicamentos , Femenino , Humanos , Ifosfamida/efectos adversos , Lactante , MasculinoRESUMEN
Children with B cell non-Hodgkin's lymphoma who have not relapsed 1 year after diagnosis and treatment are generally cured. We report here the results of treatment in 114 children who all had a minimum follow-up of 20 months. The protocol LMB 0281 from the French Pediatric Oncology Society was used. This nine-drug intensive-pulsed chemotherapy was based on high-dose cyclophosphamide, high-dose methotrexate (HD MTX), and cytosine arabinoside (ara-C) in continuous infusion. CNS prophylaxis was with chemotherapy only. No local irradiation was performed. No debulking surgery was recommended. There were 72 patients with stage III lymphoma and 42 patients with stage IV lymphoma or B cell acute lymphocytic leukemia (B-ALL). Among those 42 patients, seven had CNS involvement alone, 21 had bone marrow alone, and 14 had both; 26 had greater than 25% blast cells in bone marrow, 14 of whom had blast cells in blood. The primary site of involvement was the abdomen in 90 patients, the Waldeyer Ring in nine, and various sites in eight; seven patients presented without tumor. Seventy-seven patients are alive with a median follow-up of 2 years and 8 months. Seven patients died due to initial treatment failure, 11 died from toxicity, and 19 died after relapse. Among the 93 patients without initial CNS involvement, only one isolated relapse in CNS occurred. Survival and disease-free survival rates reached 67% and 64%, respectively, for all patients, 75% and 73% for stage III patients and 54% and 48% for stage IV and B-ALL patients. Bone marrow involvement was not an adverse prognostic factor. Contrary initial CNS involvement indicated a bad prognosis with a disease-free survival rate of 19% compared with 76% without CNS disease. This study showed that CNS prophylaxis and local control of the primary tumor can be achieved by intensive chemotherapy alone, without radiotherapy or debulking surgery.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Linfocitos B/patología , Leucemia Linfoide/tratamiento farmacológico , Linfoma/tratamiento farmacológico , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Linfocitos B/inmunología , Enfermedades de la Médula Ósea/tratamiento farmacológico , Niño , Preescolar , Ensayos Clínicos como Asunto , Esquema de Medicación , Femenino , Enfermedades Hematológicas/inducido químicamente , Humanos , Leucemia Linfoide/sangre , Leucemia Linfoide/mortalidad , Linfoma/sangre , Linfoma/mortalidad , Masculino , Estadificación de Neoplasias , Neoplasias del Sistema Nervioso/tratamiento farmacológico , Virosis/inducido químicamenteRESUMEN
PURPOSE: To clarify treatment strategy for lymphocyte-predominant Hodgkin's lymphoma (LPHL), the French Society of Pediatric Oncology initiated a prospective, nonrandomized study in 1988. Patients received either standard treatment for Hodgkin's lymphoma or were not treated beyond initial adenectomy. PATIENTS AND METHODS: From 1988 to 1998, 27 patients were available for study. Twenty-four patients were male, and median age was 10 years (range, 4 to 16 years). Twenty-two, two, and three patients had stage I, II, and III disease, respectively. Thirteen patients (stage I, n = 11; stage III, n = 2) received no further treatment after initial surgical adenectomy (SA). Fourteen patients received combined treatment (CT; n = 10), involved-field radiotherapy alone (n = 1), or chemotherapy alone (n = 3). The two groups were comparable for clinical status, treatment, and follow-up. RESULTS: Twenty-three of 27 patients achieved complete remission (CR). With a median follow-up time of 70 months (range, 32 to 214 months), overall survival to date is 100%, and overall event-free survival (EFS) is 69% +/- 10% (SA, 42% +/- 16%; CT, 90% +/- 8.6%; P <.04). If we considered only the patients in CR after initial surgery (n = 12), EFS was no longer significantly different between the two groups. Patients with residual mass after initial surgery (n = 15) had worse EFS if they did not receive complementary treatment (P <.05). CONCLUSION: Although based on a small number of patients, our study showed that (1). no further therapy is a valid therapeutic approach in LPHL patient in CR after initial lymph node resection, and (2). complementary treatment diminishes relapse frequency but has no impact on survival.
Asunto(s)
Enfermedad de Hodgkin/terapia , Escisión del Ganglio Linfático , Adolescente , Niño , Preescolar , Terapia Combinada , Femenino , Enfermedad de Hodgkin/patología , Humanos , Masculino , Recurrencia Local de Neoplasia , Estudios Prospectivos , Inducción de Remisión , Análisis de Supervivencia , Resultado del TratamientoRESUMEN
PURPOSE: To evaluate growth, thyroid function, puberty, cardiac function, and the incidence of cataracts in children who received allogeneic bone marrow transplantation (BMT) for acute myeloblastic leukemia (AML) in first complete remission (CR) after a preparation with or without total-body irradiation (TBI). PATIENTS AND METHODS: Among 45 children studied, 26 received busulfan-cyclophosphamide (Bu-Cy) in preparation for transplantation and 19 received TBI. TBI was fractionated in nine cases and delivered as a single dose in 10. Four children in the Bu-Cy group and none in the TBI group had received prior cranial radiation. The mean follow-up duration after BMT was 5.9 years for the whole group. RESULTS: The mean cumulative changes in height SD score (SDS) were -0.86 at 3 years and -1.56 at 5 years in the TBI group, whereas these changes were only -0.05 and -0.17 in the Bu-Cy group (P < .01 at 3 and 5 years). The 6-year probability of hypothyroidism was 9% +/- 8% in the Bu-Cy group and 43% +/- 15% after TBI (P < .02). Pubertal development after Bu-Cy was assessable in two girls and five boys: both girls had primary ovarian failure, whereas Leydig cell function appeared to be preserved in the five boys. One child who had received anthracycline when he was less than 1 year old developed cardiac dysfunction 4 years after Bu-Cy. The 6-year probability of cataracts was 70% +/- 13% in the TBI group and 0% after Bu-Cy. CONCLUSION: The use of Bu-Cy represents an alternative transplant cytoreductive regimen for children with AML in first CR, which can reduce the risk of posttransplant growth impairment, thyroid dysfunction, Leydig cell damage, and the incidence of cataracts.
Asunto(s)
Trasplante de Médula Ósea/efectos adversos , Leucemia Mieloide Aguda/terapia , Pubertad/efectos de los fármacos , Acondicionamiento Pretrasplante/métodos , Adolescente , Antineoplásicos Alquilantes/administración & dosificación , Antineoplásicos Alquilantes/efectos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/administración & dosificación , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Busulfano/administración & dosificación , Busulfano/efectos adversos , Niño , Preescolar , Ciclofosfamida/administración & dosificación , Ciclofosfamida/efectos adversos , Supervivencia sin Enfermedad , Femenino , Crecimiento/efectos de los fármacos , Crecimiento/efectos de la radiación , Humanos , Lactante , Leucemia Mieloide Aguda/radioterapia , Masculino , Irradiación Corporal TotalRESUMEN
PURPOSE: To determine whether the use of a recombinant human granulocyte colony-stimulating factor ([G-CSF] lenogastrim) can increase the chemotherapy dose-intensity (CDI) delivered during consolidation chemotherapy of childhood acute lymphoblastic leukemia (ALL). PATIENTS AND METHODS: Sixty-seven children with very high-risk ALL were randomized (slow early response to therapy, 55 patients; translocation t(9;22) or t(4;11), 12 patients). Consolidation consisted of six courses of chemotherapy; the first, third, and fifth courses were a combination of high-dose cytarabine, etoposide, and dexamethasone (R3), whereas the second, fourth, and sixth courses included vincristine, prednisone, cyclophosphamide, doxorubicin, and methotrexate (COPADM). G-CSF was given after each course, and the next scheduled course was started as soon as neutrophil count was > 1 x 10(9)/L and platelet count was > 100 x 10(9)/L. CDI was calculated using the interval from day 1 of the first course to hematologic recovery after the fifth course (100% CDI = 105-day interval). RESULTS: CDI was significantly increased in the G-CSF group compared with the non-G-CSF group (mean +/- 95% confidence interval, 105 +/- 5% v 91 +/- 4%; P <.001). This higher intensity was a result of shorter post-R3 intervals in the G-CSF group, whereas the post-COPADM intervals were not statistically reduced. After the R3 courses, the number of days with fever and intravenous antibiotics and duration of hospitalization were significantly decreased by G-CSF, whereas reductions observed after COPADM were not statistically significant. Duration of granulocytopenia was reduced in the G-CSF group, but thrombocytopenia was prolonged, and the number of platelet transfusions was increased. Finally, the 3-year probability of event-free survival was not different between the two groups. CONCLUSION: G-CSF can increase CDI in high-risk childhood ALL. Its effects depend on the chemotherapy regimen given before G-CSF administration. In our study, a higher CDI did not improve disease control.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Factor Estimulante de Colonias de Granulocitos/uso terapéutico , Neutropenia/inducido químicamente , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Trombocitopenia/inducido químicamente , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Niño , Preescolar , Ciclofosfamida/administración & dosificación , Citarabina/administración & dosificación , Dexametasona/administración & dosificación , Relación Dosis-Respuesta a Droga , Doxorrubicina/administración & dosificación , Etopósido/administración & dosificación , Femenino , Factor Estimulante de Colonias de Granulocitos/farmacología , Humanos , Lactante , Recién Nacido , Masculino , Metotrexato/administración & dosificación , Neutropenia/prevención & control , Prednisona/administración & dosificación , Proteínas Recombinantes , Trombocitopenia/prevención & control , Resultado del Tratamiento , Vincristina/administración & dosificaciónRESUMEN
Pediatric experience with extracorporeal photochemotherapy (ECP) for graft-versus-host disease (GvHD) has mainly been reported by Italian and French groups. Data concerning 41 children with acute GvHD and 63 children affected by chronic GvHD are available. In 73 and 63% of them, respectively, improvement was observed, with addition of ECP to their immunosuppressive regimen. Treatment with ECP was associated with minimal side effects, even in the smallest of patients. In all responded pediatric patients, both with acute and chronic GvHD, ECP allowed progressive reduction or discontinuation of the concomitant pharmacological immunosuppressive therapy without an increase in GvHD activity. These data show that ECP is a useful therapy for children affected by GvHD resistant to conventional treatment and can be safely used.
Asunto(s)
Enfermedad Injerto contra Huésped/terapia , Fotoféresis , Niño , Preescolar , Femenino , Humanos , Terapia de Inmunosupresión/métodos , Lactante , Masculino , Fotoféresis/métodosRESUMEN
Using appropriate DNA probes, the configurations of the T-cell receptor beta-chain genes and immunoglobulin heavy-chain genes were studied in patients diagnosed as having the following malignancies: 7 chronic myeloid leukemia, 13 acute myeloblastic leukemia, 9 acute lymphocytic leukemia and 20 chronic lymphocytic leukemia. Rearrangements not corresponding to the immunotype were unexpectedly found in lineage neoplasias.
Asunto(s)
Reordenamiento Génico de la Cadena beta de los Receptores de Antígenos de los Linfocitos T , Reordenamiento Génico , Genes de Inmunoglobulinas , Leucemia/genética , Linfoma de Burkitt/sangre , Linfoma de Burkitt/genética , Genotipo , Humanos , Inmunofenotipificación , Leucemia/sangre , Leucemia Linfocítica Crónica de Células B/sangre , Leucemia Linfocítica Crónica de Células B/genética , Leucemia Prolinfocítica de Células T/sangre , Leucemia Prolinfocítica de Células T/genética , Leucemia-Linfoma de Células T del Adulto/sangre , Leucemia-Linfoma de Células T del Adulto/genéticaRESUMEN
Adenosine deaminase (ADA) and purine nucleoside phosphorylase (PNP) were measured in normal human and in malignant lymphoid cells. Thymocytes had high ADA activity (21.2 +/- 6.8 10(3) nM/h/mg) and low PNP activity (1.2 +/- 0.6), whereas T peripheral blood lymphocytes (PBL) had low ADA activity (1.20 +/- 0.22) and high PNP activity (2.8 +/- 1.3). Moreover cortico-thymocytes had higher ADA and lower PNP levels than medullary thymocytes. A linear correlation was observed between ADA and PNP activities in both thymocytes and T-PBL. Cells from 13 patients with T acute lymphoblastic leukemia (ALL) and 10 patients with T lymphoblastic lymphoma (LL) had very high levels of ADA (respectively 13.0 +/- 5.4 and 22.8 +/- 14) and low levels of PNP (respectively 1.9 +/- 0.8 and 2.5 +/- 1.4). However no clear relationship appeared between subgroups of these T-cell malignancies defined by their patterns of surface antigens, revealed by reactivity with monoclonal antibodies, and ADA and PNP levels, and there was no correlation between the two enzymes. In contrast, cells from 31 patients with HLA-DR+ common ALL had significantly low values of ADA as compared to cells from six patients with HLA-DR- common ALL and a linear correlation was observed between ADA and PNP in cells from children with non-T, non-B ALL. These results show that specific stages of T-cell development may be characterized by the relationships and the correlation between the two enzymes and suggest that T-ALL and T-LL appear to be the group of lymphoid malignancies with a high degree of incoordination between ADA and PNP activities.
Asunto(s)
Adenosina Desaminasa/metabolismo , Leucemia/enzimología , Nucleósido Desaminasas/metabolismo , Pentosiltransferasa/metabolismo , Purina-Nucleósido Fosforilasa/metabolismo , Linfocitos T/enzimología , Antígenos de Superficie/análisis , Antígenos HLA-DR , Antígenos de Histocompatibilidad Clase II/análisis , Humanos , Leucemia/clasificación , Timo/enzimologíaRESUMEN
In four children from the same family, we have observed an association of mental retardation, dwarfism, hypertelorism, facial clefting and urogenital abnormalities. Clinical and laboratory data suggest that it is a previously undescribed genetic syndrome, ie, a pleiotropic autosomal recessive trait.