RESUMEN
BACKGROUND: Asparaginase is essential for treating T-cell acute lymphoblastic leukemia (T-ALL). Despite the ongoing debate on whether T-ALL and T-cell lymphoblastic lymphoma (T-LBL) are the same disease entity or two distinct diseases, patients with T-LBL often receive the same or similar treatment protocols as those with T-ALL. METHODS: The outcomes of patients with or without L-asparaginase discontinuation were retrospectively analyzed among four national protocols: Japan Association of Childhood Leukemia Study (JACLS) ALL-02 and ALL-97 for T-ALL and Japanese Pediatric Leukemia/Lymphoma Study Group ALB-NHL03 and JACLS NHL-98 for T-LBL. The hazard ratio (HR) was calculated with the Cox regression model by considering L-asparaginase discontinuation as a time-dependent variable. RESULTS: In total, 199 patients with T-ALL, and 133 patients with T-LBL were included. L-asparaginase discontinuation compromised event-free survival (EFS) of T-ALL patients (ALL-02: HR 3.32, 95% confidence interval [CI] 1.40-7.90; ALL-97: HR 3.39, 95%CI 1.19-9.67). Conversely, EFS compromise was not detected among T-LBL patients (ALB-NHL03: HR 1.39, 95%CI 0.41-4.68; NHL-98: HR 0.92, 95%CI 0.11-7.60). CONCLUSION: The effects of L-asparaginase discontinuation differed between T-ALL and T-LBL. We assume that the differential impact results from (1) the inherent differential response to L-asparaginase between them and/or (2) a less stringent assessment of early treatment response in T-LBL than in T-ALL. Given the poor salvage rate of refractory or relapsed T-ALL and T-LBL, optimization of the frontline therapy is critical, and the current study provides a new suggestion for further treatment modifications. However, larger studies in contemporary intensified treatment protocols are required.
Asunto(s)
Asparaginasa , Leucemia-Linfoma Linfoblástico de Células T Precursoras , Humanos , Asparaginasa/uso terapéutico , Asparaginasa/administración & dosificación , Niño , Masculino , Femenino , Leucemia-Linfoma Linfoblástico de Células T Precursoras/tratamiento farmacológico , Leucemia-Linfoma Linfoblástico de Células T Precursoras/mortalidad , Preescolar , Estudios Retrospectivos , Adolescente , Lactante , Resultado del Tratamiento , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Japón , Antineoplásicos/uso terapéuticoRESUMEN
Pediatric patients with lymphoblastic lymphoma are generally treated using the Berlin-Frankfurt-Munster (BFM) 90 protocol, which is the standard treatment strategy for pediatric acute lymphoblastic leukemia, and have a favorable outcome. However, this intense regimen includes high total doses of anthracycline and alkylating agents, and is known to cause late complications. We therefore planned a clinical trial to examine the efficacy and safety of a modified BFM regimen. We expect that this phase II, nationwide multicenter trial will help to establish an effective and safer standard therapy for stage I/II pediatric lymphoblastic lymphoma.
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Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Ensayos Clínicos Fase II como Asunto , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Adolescente , Asparaginasa , Niño , Ciclofosfamida , Citarabina , Dexametasona , Doxorrubicina , Humanos , Estudios Multicéntricos como Asunto , Tioguanina , VincristinaRESUMEN
This trial enrolls patients with untreated Hodgkin's lymphoma aged<20 years at diagnosis and examines the effects of omitting radiation therapy if the FDG-positron emission tomography (PET) findings after two completed cycles of combination chemotherapy are negative. It thereby aims to determine whether patients who truly require radiation therapy can be identified by FDG-PET. If so, this modality could be used to omit radiation therapy for all other patients, decreasing the risk of serious long-term complications without affecting survival rates. The outcomes of patients for whom FDG-PET is used to assess early treatment response will also be determined.
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Ensayos Clínicos Fase II como Asunto , Fluorodesoxiglucosa F18 , Enfermedad de Hodgkin/tratamiento farmacológico , Tomografía de Emisión de Positrones/métodos , Niño , Femenino , Enfermedad de Hodgkin/diagnóstico por imagen , Enfermedad de Hodgkin/mortalidad , Humanos , Masculino , Estudios Multicéntricos como Asunto , Estudios RetrospectivosRESUMEN
We reviewed the immunophenotypic subtypes of pediatric T-cell lymphoblastic lymphoma in the Japanese Pediatric Leukemia/Lymphoma Study Group ALB-NHL03 study. Of the 104 patients, 40 patients each had sufficient data to evaluate the immunophenotypes and early T-cell precursor (ETP) subtype. Pro-T, pre-T, intermediate T, and mature T cells were observed in 1, 9, 21, and 9 cases, respectively. The 3-year event-free survival (EFS) rates of those with pro-T/pre-T, intermediate T, and mature T cells were 80.0±12.6%, 71.4±9.9%, and 88.9±10.5%, respectively (P=0.546). There were 8 and 32 cases of ETP and non-ETP subtypes, with 3-year EFS rates of 75.0±15.3% and 71.9±8.0%, respectively (P=0.828), indicating that the immunophenotypic subtype was not predictive of EFS in this study.
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Células Precursoras de Linfocitos T/patología , Leucemia-Linfoma Linfoblástico de Células T Precursoras/clasificación , Pronóstico , Niño , Femenino , Humanos , Inmunofenotipificación , Japón , Masculino , Leucemia-Linfoma Linfoblástico de Células T Precursoras/mortalidad , Leucemia-Linfoma Linfoblástico de Células T Precursoras/patología , Estudios Retrospectivos , Análisis de Supervivencia , Subgrupos de Linfocitos T/patologíaRESUMEN
Follicular lymphoma (FL) is quite rare in children. There have been only two major reports on pediatric FL. The present retrospective study on pediatric FL in Japan, including FL with diffuse large B cell lymphoma (DLBCL), analyzed data from 1991 to 2014. Twenty-two patients with pediatric FL were analyzed. Sixteen patients were boys and six were girls. Median age of onset was 9 years (range 4-17 years). In 11 patients, DLBCL co-existed with FL. The initial lesions involved cervical lesions in 16 patients, and the abdomen in six. With regard to stage of disease at diagnosis, 17 patients were at stage I or II, four were at stage III, and one was at stage IV. Chemotherapy was administered in 18 patients, and only resection was performed in four patients. Mature B lymphoma regimens were selected for 17 patients who received chemotherapy. Although two patients relapsed, all patients are currently alive and disease free. The median follow-up period was 54.5 months (range 6-126 months). Patients having FL with DLBCL were younger compared with those having FL, and this disease was more frequently observed in female patients. Our data revealed that FL in Japanese children is a tumor with good prognosis, as in reports from the United States and Europe.
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Linfoma Folicular/epidemiología , Linfoma de Células B Grandes Difuso/epidemiología , Adolescente , Factores de Edad , Niño , Preescolar , Femenino , Humanos , Japón/epidemiología , Linfoma Folicular/diagnóstico , Linfoma Folicular/terapia , Linfoma de Células B Grandes Difuso/diagnóstico , Linfoma de Células B Grandes Difuso/terapia , Masculino , Estadificación de Neoplasias , Pronóstico , Estudios Retrospectivos , Factores SexualesRESUMEN
Reversible cerebral vasoconstriction syndrome (RCVS) is a syndrome characterized by severe headache with segmental vasoconstriction of the cerebral arteries that resolves within 12 weeks. A 16-year-old girl with refractory cytopenia of childhood, who was receiving the immunosuppressant cyclosporine, developed severe headache and was diagnosed with RCVS using magnetic resonance imaging, including magnetic resonance angiography (MRA). MRA is a non-invasive and very effective technique for diagnosing RCVS. MRA should be performed at the onset of severe headache during immunosuppressant administration for children with hematological disorders and may prevent sequelae such as posterior reversible encephalopathy syndrome or ischemic attack.
RESUMEN
The purpose of this study was to explore ways of enhancing psychosocial strengths in newly diagnosed and relapsed adolescents and young adults (AYAs) to improve their resilience. A descriptive case study was used. The adolescent resilience model (ARM) and the self-sustaining process model were applied as theories. The data were analyzed using pattern-matching logic. Semistructured interviews were conducted with 18 patients aged 12 to 24 years and discharged within 10 years. We found that the newly diagnosed and the relapsing AYAs developed the 5 strength factors of the ARM during and after treatment. Whether the individuals cultivated a positive attitude and sense of purpose early or late, the AYAs developed resilience eventually. A positive attitude and sense of purpose during the early phase of care may be essential for improving resilience. The AYAs benefited from the support of their parents, friends, and previous experience. Individualized support and social resources may be important to develop these strengths. Further research is needed to develop strengths and improve resilience in newly diagnosed AYAs.
Asunto(s)
Adaptación Psicológica , Acontecimientos que Cambian la Vida , Neoplasias/psicología , Autocuidado/psicología , Apoyo Social , Adolescente , Ansiedad , Actitud Frente a la Salud , Femenino , Humanos , Japón/epidemiología , Masculino , Neoplasias/epidemiología , Padres , Resiliencia Psicológica , Adulto JovenRESUMEN
BACKGROUND: Childhood advanced lymphoblastic lymphoma (LBL) has a favorable outcome with an event-free survival (EFS) rate of over 80% in response to treatment strategies for acute lymphoblastic leukemia (ALL). However, no progress has been made in this outcome over the past 10 years. PROCEDURE: We conducted the first nationwide prospective study of childhood advanced LBL to assess the efficacy and safety of ALL-directed therapy with an intensified maintenance phase. We omitted local radiotherapy including prophylactic cranial radiotherapy except for patients with initial central nervous system disease. The total duration of the treatment was 24 months. RESULTS: For the 136 patients analyzed in this study, 5-year overall survival (OS) was 82.9% and 5-year EFS was 77.9%. Thirty events were observed and 14 occurred before the initiation of intensified maintenance phase. Of 14 events, nine were observed as mediastinal enlargement. There was no significant difference in outcome when stratified according to gender or by immunophenotype. The 5-year EFS according to clinical stage in patients with T-cell LBL (T-LBL) was 70.6% for stage III and 88.9% for stage IV (P = 0.037). CONCLUSIONS: Our first nationwide study provided about 80% cure rate with only one case of toxic death in childhood advanced LBL. However, our intensified maintenance therapy could not improve the survival outcome. There was a trend of better EFS in Japanese patients with T-LBL stage IV than T-LBL stage III.
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Quimioterapia de Mantención/métodos , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamiento farmacológico , Adolescente , Niño , Preescolar , Supervivencia sin Enfermedad , Femenino , Humanos , Lactante , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidad , Pronóstico , Estudios Prospectivos , Insuficiencia del Tratamiento , Resultado del TratamientoRESUMEN
The objective of this study was to assess the impact of the primary prophylaxis of granulocyte colony-stimulating factor (G-CSF) in the management of childhood B-cell non-Hodgkin lymphoma (B-NHL). Patients with advanced-stage mature B-NHL were randomized to receive prophylactic G-CSF (G-CSF+) or not receive G-CSF (G-CSF-) based on protocols of the B-NHL03 study. The G-CSF group received 5 µg/kg/d Lenograstim from day 2 after each course of six chemotherapy courses. Fifty-eight patients were assessable, 29 G-CSF + and 29 G-CSF-. G-CSF + patients showed a positive impact on the meantime to neutrophil recovery and hospital stay. On the other hand, they had no impact in the incidences of febrile neutropenia, serious infections, stomatitis and total cost. Our study showed that administration of prophylactic G-CSF through all six chemotherapy courses for childhood B-NHL showed no clinical and economic benefits for the management of childhood B-NHL treatment.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/efectos adversos , Quimioprevención , Factor Estimulante de Colonias de Granulocitos/uso terapéutico , Linfoma de Células B/complicaciones , Neutropenia/etiología , Neutropenia/prevención & control , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Niño , Preescolar , Femenino , Factor Estimulante de Colonias de Granulocitos/administración & dosificación , Factor Estimulante de Colonias de Granulocitos/efectos adversos , Costos de la Atención en Salud , Hospitalización , Humanos , Infecciones/etiología , Linfoma de Células B/diagnóstico , Linfoma de Células B/tratamiento farmacológico , Masculino , Metástasis de la Neoplasia , Estadificación de Neoplasias , Trombocitopenia/etiología , Resultado del TratamientoRESUMEN
Results of pediatric lymphoma treatment have improved markedly over the past 30 years. In Hodgkin's lymphoma, the 5 year event-free survival (EFS) was 81.5% in a retrospective study. In the ALB-NHL03 study, the 5 year EFS according to clinical stage in patients with lymphoblastic T-cell lymphoma (T-LBL) was 70.6% for stage III and 88.9% for stage IV. In mature B-cell lymphoma, the B-NHL03 study indicated that the 4 year EFS according to treatment group was 94% for group 1, 98% for group 2, 84% for group 3, and 78% for group 4. Moreover, the 2 year EFS rate was 81% in Japanese advanced stage patients based on the international ALCL99 study. Thus, EFS >80% was achieved in any subtype of pediatric lymphoma. With regard to refractory or recurrent lymphoma, however, treatment methods for improvement of the survival rate in these patients still need to be developed. Also the difference between child, and adolescent and young adult patients still needs to be clarified, and treatment protocols developed. Although lymphoma treatment does not greatly change according to country, it does differ between other countries and Japan for some subtypes of lymphoma. In particular, the results of treatment of stage III T-LBL in Japan are worse than those in the USA and Europe. The priority in future studies will be to collect data on these differences, and the reasons for these differences.
Asunto(s)
Predicción , Planificación en Salud/tendencias , Linfoma/terapia , Niño , Terapia Combinada , Humanos , Japón/epidemiología , Linfoma/epidemiología , Morbilidad/tendenciasRESUMEN
Little information is available on cytogenetic abnormalities and their prognostic importance in childhood mature B-cell non-Hodgkin lymphoma (B-NHL). We performed a review of 79 abnormal karyotypes in childhood B-NHL treated by a uniform protocol. Del(17p) was independently associated with significantly inferior event-free survival in Burkitt or Burkitt-like lymphoma. The adverse prognosis of MYC/8q24 rearrangement, +7q or del(13q), was not observed, which had been suggested as risk factors in FAB/LMB96. Our results imply the possible existence of a biological difference among ethnicities and should be useful to narrow down the gene causing poor prognosis in childhood B-NHL.
Asunto(s)
Aberraciones Cromosómicas , Linfoma de Células B/genética , Linfoma de Células B/mortalidad , Proteínas Proto-Oncogénicas c-myc/genética , Adolescente , Niño , Preescolar , Terapia Combinada , Femenino , Estudios de Seguimiento , Humanos , Linfoma de Células B/terapia , Masculino , Estadificación de Neoplasias , Pronóstico , Tasa de SupervivenciaRESUMEN
BACKGROUND: Plasma monitoring of Methotrexate (MTX) levels is a standard approach to predict MTX-related toxicities in a high-dose (HD) MTX monotherapy for childhood acute lymphoblastic leukemia. However, it is uncertain whether plasma MTX levels can predict MTX-related toxicity in the HDMTX plus additional chemotherapy for childhood B-cell nonHodgkin lymphoma (B-NHL). PROCEDURES: To statistically analyze the relationship between MTX pharmacokinetic parameters and MTX-related toxicities, we collected data from patients with delayed MTX elimination (≥1 µM at 48 hr and/or ≥0.5 µM at 72 hr) in the Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG) BNHL 03 study. Blood MTX levels were measured at 24, 48, and 72 hr after 3 or 5 g/m2 HD-MTX administration for 24 hr. RESULTS: Three hundred and four patients received 2-4 courses of the HDMTX plus additional chemotherapy, and delayed MTX elimination was observed in 165 courses of 127 patients. In those, nephrotoxicity was significantly correlated with plasma MTX levels for each patient (P = 0.03), and also for each course (P = 0.009), but no other toxicities were correlated. Another analysis according to HDMTX courses showed no significant correlation between the first high plasma MTX levels and subsequent MTX levels in later course. It also showed that incidence of liver and gastrointestinal toxicities was most frequent in the first HDMTX course, and then sharply decreased in later courses (P < 0.001). CONCLUSIONS: Our results suggest that plasma MTX level is not a reliable predictor for adverse events except for nephrotoxicity in multiple HDMTX therapy courses in childhood B-NHL. Pediatr Blood Cancer 2015;62:279-284. © 2014 Wiley Periodicals, Inc.
Asunto(s)
Lesión Renal Aguda/inducido químicamente , Antimetabolitos Antineoplásicos/toxicidad , Enfermedad Hepática Inducida por Sustancias y Drogas/diagnóstico , Linfoma no Hodgkin/tratamiento farmacológico , Metotrexato/toxicidad , Lesión Renal Aguda/sangre , Antimetabolitos Antineoplásicos/sangre , Antimetabolitos Antineoplásicos/uso terapéutico , Enfermedad Hepática Inducida por Sustancias y Drogas/sangre , Femenino , Humanos , Riñón/efectos de los fármacos , Hígado/efectos de los fármacos , Masculino , Metotrexato/sangre , Metotrexato/uso terapéuticoRESUMEN
Post-transplant ES, which is often resistant to therapies, has seldom been described. This report describes a case of ES after UBMT for RCC. A five-yr-old boy developed RCC with no evidence of monosomy 7. Because no matching family donors were available for SCT and immunosuppressive therapy was ineffective, UBMT was performed when he was six yr old. The conditioning regimen included TAI (3 Gy) and administration of FLU, CY, and rabbit antithymocyte globulin. The recovery of blood cells was good. He displayed grade II acute GVHD involving only the skin. ES developed on day 66, with positive results for Epstein-Barr virus DNA and HHV 6. Cytopenia was resolved with treatment with RTX, GCV, an escalated dose of steroids, high-dose gammaglobulin, and romiplostim. No relapse has occurred since discontinuing steroids on day 177 and romiplostim on day 268. Post-SCT ES after UBMT is rare, and the risk factors and therapies are unclear. Prospective analysis and collection of cases from multiple centers are required for clarification.
Asunto(s)
Anemia Hemolítica Autoinmune/etiología , Trasplante de Médula Ósea , Púrpura Trombocitopénica Idiopática/complicaciones , Púrpura Trombocitopénica Idiopática/terapia , Trombocitopenia/etiología , Preescolar , ADN/análisis , ADN Viral/metabolismo , Herpesvirus Humano 4 , Humanos , Inmunosupresores/efectos adversos , Inmunosupresores/uso terapéutico , Masculino , Estudios Prospectivos , Receptores Fc/química , Receptores Fc/uso terapéutico , Proteínas Recombinantes de Fusión/química , Proteínas Recombinantes de Fusión/uso terapéutico , Factores de Riesgo , Esteroides/uso terapéutico , Trombopoyetina/química , Trombopoyetina/uso terapéutico , Factores de Tiempo , Resultado del Tratamiento , gammaglobulinas/química , gammaglobulinas/uso terapéuticoRESUMEN
BACKGROUND: Previous Japanese studies of childhood B-cell non-Hodgkin lymphoma (B-NHL) have shown a favorable outcome, though the study size was too small to effectively assess the efficacy and safety of treatment for childhood B-NHL. PROCEDURE: We performed a nation-wide prospective B-NHL03 study to assess the efficacy and safety of short-pulse intensive chemotherapy for children with B-NHL. They were stratified into four treatment groups according to disease stage, tumor resectability and bone marrow/CNS involvement: Group 1 with all resected stage I/II, Group 2 with non-resected stage I/II, Group 3 with stage III & CNS-negative stage IV, and Group 4 with CNS-positive stage IV & Burkitt leukemia. Treatment duration was 2 courses for Group 1, 4 courses for Group 2, and 6 courses for Groups 3 and 4, respectively. CNS irradiation was omitted in all patients. RESULTS: The follow-up time ranged from 0.8 to 88 months, with a median of being 45 months. For 321 patients analyzed in this study, overall survival and event-free survival (EFS) at 4 years was 92.7% and 87.4%, respectively. The 4-year EFS according to treatment group were 94% for Group 1 (n = 17), 98% for Group 2 (n = 103), 84% for Group 3 (n = 111), and 78% for Group 4 (n = 90). There was no significant difference in outcome by histology. Therapy-related death occurred in three patients in remission. CONCLUSIONS: Our nationwide large-scale study resulted in a cure rate above 90% with <1% toxic death in childhood B-NHL.
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Linfoma de Células B/mortalidad , Linfoma de Células B/patología , Linfoma de Células B/terapia , Adolescente , Pueblo Asiatico , Niño , Preescolar , Femenino , Estudios de Seguimiento , Humanos , Lactante , Recién Nacido , Japón/epidemiología , Masculino , Estudios Prospectivos , Factores de TiempoRESUMEN
Immediate allergy to l-asparaginase (ASP) is a major obstacle in treating lymphoid malignancies. ASP-specific immunoglobulin G (ASP-IgG) has been used as a surrogate marker. Recently, the CD203c-basophil activation test (BAT) was found to be useful in diagnosing IgE-mediated allergies. We compared the diagnostic utility of the CD203c-BAT to that of ASP-IgG levels in determining ASP allergies in children. Eight ASP allergic reactions occurred over 75 ASP treatment courses. The sensitivity, specificity and area under the receiver operating characteristic curve of CD203c-BAT were similar to the ASP-IgG levels (0.75 vs. 0.85, 0.82 vs. 0.78 and 0.81 vs. 0.85, respectively). Positive skin prick test results in patients with ASP allergy suggested that ASP-IgE was one of the key players in ASP allergy. A combination of the BAT with the ASP-IgG level had the highest specificity (0.95) and positive predictive value (0.62), which permitted us to identify ASP allergy more effectively.
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Asparaginasa/inmunología , Basófilos/inmunología , Basófilos/metabolismo , Hipersensibilidad Inmediata/inmunología , Hipersensibilidad Inmediata/metabolismo , Hidrolasas Diéster Fosfóricas/metabolismo , Pirofosfatasas/metabolismo , Adolescente , Biomarcadores/sangre , Biomarcadores/metabolismo , Niño , Preescolar , Femenino , Humanos , Hipersensibilidad Inmediata/diagnóstico , Inmunoglobulina E/inmunología , Inmunoglobulina G/sangre , Inmunoglobulina G/inmunología , Lactante , Linfoma/complicaciones , Linfoma/terapia , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicaciones , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Sensibilidad y EspecificidadRESUMEN
BACKGROUND: Acquired somatic mutations of JAK2 have been reported to play a pivotal role in the pathogenesis of BCR-ABL1-negative myeloproliferative neoplasm (MPN). However, the molecular characteristics of childhood MPN remain to be elucidated. PATIENT AND METHODS: We investigated a group of pediatric patients diagnosed either with essential thrombocythemia (ET; N = 9) or polycythemia vera (PV; N = 4) according to WHO criteria (median age = 10 years; range 1.5-15 years) in whom direct sequencing was performed for the existence of genetic alterations in JAK2, MPL, TET2, ASXL1, CBL, IDH1, and IDH2. More sensitive allele specific polymerase chain reaction was used for JAK2(V617F) genotyping. RESULTS: We found three patients harbor JAK2(V617F) mutation (2/9 ET and 1/4 PV). Bone marrow examination showed small and large megakaryocytes with dysplastic features in JAK2(V617F)-positive ET patients compared to those without JAK2(V617F). We identified a previously unrecognized missense mutation at codon 1230 in exon 12 of ASXL1 gene in ET and PV patients (1/9 ET and 1/4 PV). Otherwise, no genetic alterations could be detected in JAK2 exon 12, MPL, TET2, CBL, IDH1, and IDH2 in all ET and PV patients. CONCLUSION: Although JAK2 mutations in childhood ET and PV are not as frequent as reported in adult patients, JAK2 is the most frequently mutated gene in childhood MPN known so far. Owing to the presence of childhood MPN without any genetic alterations in JAK2, MPL, TET2, ASXL1, CBL, IDH1, and IDH2, new biological markers have to be found.
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Janus Quinasa 2/genética , Mutación , Policitemia Vera/genética , Trombocitemia Esencial/genética , Adolescente , Pueblo Asiatico , Niño , Preescolar , Femenino , Humanos , Lactante , MasculinoRESUMEN
T-cell acute lymphoblastic leukaemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) are combined into one category as T lymphoblastic leukaemia/lymphoma in the current World Health Organization (WHO) classification. However, there is still ongoing discussion on whether T-ALL and T-LBL are two separate entities or represent two variant phenotypes of the same disease. Cytogenetic analysis has been used to identify the molecular background of haematological malignancies. To compare the distribution of chromosomal abnormalities of T-ALL and T-LBL, large series of cytogenetic data are required, but are absent in T-LBL in contrast to the abundant data in T-ALL. Among 111 T-LBL cases in our clinical trial, we obtained complete cytogenetic data from 56 patients. The comparison between our cytogenetic findings and those from three published T-LBL studies revealed no significant difference. However, meta-analysis showed that translocations involving chromosome region 9q34 were significantly more common in T-LBL than in T-ALL. In particular, four out of the 92 T-LBL cases, but none of the 523 paediatric T-ALL cases, showed translocation t(9;17)(q34;q22-23) (P=0·0004). Further studies are needed for the possible linkage between abnormal expression of genes located at 9q34 and/or 17q22-23 and the unique 'lymphoma phenotype' of T-LBL.
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Aberraciones Cromosómicas , Linfoma de Células T/genética , Leucemia-Linfoma Linfoblástico de Células T Precursoras/genética , Adolescente , Niño , Preescolar , Humanos , LactanteRESUMEN
The present study aimed to identify optimal treatment intensity in children with mosaic Down syndrome (DS) and acute megakaryoblastic leukemia (AMKL). A retrospective review of AMKL patients was undertaken to identify mosaic DS children. Between November 1992 and November 2007, seven children were diagnosed as mosaic DS and AMKL. The median age at diagnosis was 29 months (range 4-34 months). Three patients had a past history of transient abnormal myelopoiesis. UPN1-4 were treated with intermediate-dose cytarabine and UPN4 received additional one course of high-dose cytarabine. All of these patients were remained in first CR. UPN5-7 were treated with high-dose cytarabine according to the AML99 protocol. UPN5 with GATA1 mutation suffered from acute pneumonia and pancreatitis and discontinued chemotherapy. UPN7 relapsed after cessation of chemotherapy and was rescued with allo-PBSCT. The cumulative doses of cytarabine were 3.5-10.65 g/m(2) in the UPN1-4 and 40.4-78.4 g/m(2) in the UPN5-7. The 8-year overall survival was 100% and the 8-year event-free survival 85.7%, respectively. Our retrospective study reveals that patients with mosaic DS and AMKL have a good prognosis. Reduction in intensity may work in patients with mosaic DS as well as with AML-DS.
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Antimetabolitos Antineoplásicos/administración & dosificación , Citarabina/administración & dosificación , Síndrome de Down/genética , Síndrome de Down/mortalidad , Leucemia Megacarioblástica Aguda , Mosaicismo , Preescolar , Supervivencia sin Enfermedad , Relación Dosis-Respuesta a Droga , Femenino , Factor de Transcripción GATA1/genética , Humanos , Lactante , Leucemia Megacarioblástica Aguda/tratamiento farmacológico , Leucemia Megacarioblástica Aguda/genética , Leucemia Megacarioblástica Aguda/mortalidad , Masculino , Prevalencia , Estudios Retrospectivos , Tasa de SupervivenciaRESUMEN
BACKGROUND: Reports of non-anaplastic peripheral T-cell lymphoma (PTCL) in pediatric patients are relatively rare. PROCEDURE: We performed a retrospective analysis in patients with PTCL over an 18-year period (1991-2008). RESULTS: We could analyze clinical data in 21 patients with non-anaplastic PTCL; 10 were female and 10 male. Median age of onset was 11 years (range: 1-21 years). There were nine patients with PTCL, not otherwise specified (PTCL-NOS); ten with extranodal NK/T-cell lymphoma, nasal type; one with angioimmunoblastic T-cell lymphoma; and one with subcutaneous panniculitis-like T-cell lymphoma. Initial lesions involved cervical lymph nodes in five patients, and the skin in five patients. In five patients, hemophagocytic syndrome (HPS) was the initial clinical feature. There were 12 patients with advanced stage disease (stages III and IV). Chemotherapy and radiation was administered in 18 and 2 patients, respectively. Among the two patients who did not receive chemotherapy and radiation, one patient died while being treated for HPS but another improved spontaneously. Although 5 patients relapsed, 18 of 21 patients remained alive without disease at last follow-up. Five-year overall survival rate was 85.2%. CONCLUSIONS: Generally, the outcome results of conventional chemotherapy for high-risk PTCL are poor in adult patients. However, the excellent results in our study suggest that PTCL of childhood is quite different from that of adulthood. Although this study is first report about PTCL of Asian children, the number of patients was small in this study. Larger studies are needed to confirm these findings.