My aching bones: skeletal complications of acute lymphoblastic leukemia.
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Adolescent↗
Biomedical subjects
James B Nachman
Publications and source records attributed to James B Nachman.
Extracorporeal membrane oxygenation after stem cell transplant: clinical decision-making in the absence of evidence.
OBJECTIVE: To discuss the ethical dilemmas that arise in considering innovative therapies for critically ill children when there is little data to support their use. DESIGN: Case report of a 13-yr-old patient after autologous peripheral blood stem cell transplant for stage III neuroblastoma with sepsis and hemodynamic instability who survived to discharge after a 6-day course of extracorporeal membrane oxygenation (ECMO) support. The case serves as a source of discussion of the following: the use of available data in deciding to proceed with an unproved therapy, the approach to conversations to obtain informed consent, and the need for institutional oversight and hypothesis-driven data collection to advance pediatric critical care. SETTING: Pediatric intensive care unit at a university hospital. PATIENT: One adolescent with stage III neuroblastoma. RESULTS: Despite a lack of data to support the use of ECMO in a neutropenic oncology patient after autologous peripheral blood stem cell transplant, our patient had clinical features that suggested he was a reasonable ECMO candidate. His family gave informed consent to use ECMO and he survived. It is ethical to consider and use innovative therapies when patient characteristics are suggestive that the therapy may be successful even in the absence of evidence. This requires physicians' attention to the best interest of the patient and should occur in the setting of informed consent and rigorous data collection. CONCLUSIONS: The boundaries among standard therapy, innovative therapy, and research can be quite fluid. This case illustrates the ethical imperative to consider therapies that may be appropriate for a critically ill child even without evidence predictive of success, to have entry criteria and treatment protocols for such therapies, and to collect data from such experiences to advance the standard of care.
Adolescent↗
Adolescents with acute lymphoblastic leukemia: a new "age".
Adolescents with acute lymphoblastic leukemia (ALL) have a higher incidence of T-cell immunophenotype, a higher incidence of Philadelphia chromosome positive ALL, a lower incidence of high hyperdiploidy and TEL-AML1 translocation, and a lower incidence of extramedullary bulk disease compared to younger patients. There appears to be little difference between 10-15 and 16-21 year old patients. Adolescents with ALL have a lower event free survival (EFS) compared to younger patients. Adolescents 16-21 years of age treated on pediatric ALL trials have a significantly better EFS than those treated on adult trials. Pediatric treatment protocols utilize more vincristine, steroid and L-asparaginase compared to adult trials. In a recently completed Children's Cancer Group trial, adolescents 16-21 years of age had a four-year EFS of 73.1%. Avascular necrosis of bone is an important complication of therapy in adolescents with ALL.
Adolescent↗
Randomized comparison of low-dose involved-field radiotherapy and no radiotherapy for children with Hodgkin's disease who achieve a complete response to chemotherapy.
PURPOSE: Current standard therapy for children and adolescents with Hodgkin's disease includes combination chemotherapy and low-dose involved-field radiation (LD-IFRT). Because radiation may be associated with adverse late effects, the Children's Cancer Group (CCG) investigated whether radiation could be omitted in patients achieving a complete response to initial chemotherapy without jeopardizing the excellent outcome obtained with combined-modality therapy. PATIENTS AND METHODS: Between January 1995 and December 1998, 829 eligible patients were enrolled onto CCG 5942. A total of 501 patients who achieved an initial complete response after risk-adapted combination chemotherapy were randomized to receive LD-IFRT or no further treatment. Event-free survival (EFS) and overall survival were assessed from the date of study entry or the date of randomization, as appropriate. RESULTS: The projected 3-year EFS from study entry for the entire cohort was 87% +/- 1.2%. Among patients who achieved a complete response to initial chemotherapy, 92% +/- 1.9% of those randomized to receive LD-IFRT were alive and disease free 3 years after randomization, versus 87% +/- 2.2% for patients randomized to receive no further therapy (stratified log-rank test; P =.057). With an "as-treated" analysis, 3-year EFS after randomization for the radiation cohort was 93% +/- 1.7% versus 85% +/- 2.3% for patients receiving no further therapy (stratified log-rank test; P =.0024). Three-year survival estimates for patients treated with and without LD-IFRT were 98% +/- 1.1% for patients who received radiation and 99% +/- 0.5% for patients who did not receive radiation. CONCLUSION: LD-IFRT after an initial complete response to risk-adapted chemotherapy improved EFS. At this time, there is no survival advantage for LD-IFRT, but follow-up remains short.
Adolescent↗
Abnormalities of chromosome bands 15q13-15 in childhood acute lymphoblastic leukemia.
BACKGROUND: Recurring breakpoints in chromosome bands 15q13-15 occur infrequently in leukemia. To the authors' knowledge, the clinical significance of these breakpoints in childhood acute lymphoblastic leukemia (ALL) has not been previously investigated. METHODS: Centrally reviewed karyotypes of children with newly diagnosed ALL enrolled on Children's Cancer Group protocols from 1988 to 1995 formed the basis of the current report. Statistical analyses used chi-square tests for homogeneity of proportions, and outcome was analyzed using life table methods and associated statistics. RESULTS: Of 1946 cases with centrally reviewed and accepted cytogenetic analyses, 23 cases (1%) had breakpoints in chromosome bands 15q13-15. Most patients with 15q13-15 breakpoints had standard risk ALL, although breakpoints in 15q13-15 occurred more frequently in infants than in older children. The majority of these patients (16 patients; 70%) had balanced 15q13-15 rearrangements. Additional chromosomal abnormalities not involving 15q included abnormal 12p, abnormal 9p, Philadelphia chromosome, deletion 6q, and an 11q23 breakpoint. Thirteen (57%) 15q13-15 breakpoints occurred in pseudodiploid karyotypes; five (22%) were in hyperdiploid karyotypes with 47-50 chromosomes; two (9%) were in hyperdiploid karyotypes with > 50 chromosomes; and three (13%) were in hypodiploid karyotypes. Of the 23 patients with 15q13-15 breakpoints, 21 were survivors, 18 survived event-free for 2.2-9.3 years, and 3 were alive 1 to 3 years after a relapse at time of writing. CONCLUSIONS: The current study suggests that genes at 15q13-15 may be involved in the leukemogenesis of some cases of childhood ALL, but that with current intensive therapy such aberrations do not confer increased risk of treatment failure.
Cell Transformation, Neoplastic↗
Treatment: the most important prognostic variable.
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Antineoplastic Agents↗
Thrombocytopenia in children with severe iron deficiency.
PURPOSE: Thrombocytopenia has been reported in some children with severe iron deficiency anemia, but the validity of the association and the mechanism of the thrombocytopenia are not well established. Six children with severe iron deficiency and thrombocytopenia are described, and the literature is reviewed. PATIENTS AND METHODS: Clinical, hematologic, and morphologic data were collected and analyzed for six patients referred for evaluation of severe microcytic anemia and thrombocytopenia. RESULTS: The children ranged in age from 14 months to 17 years (median age 27 months) and were otherwise healthy. The iron deficiency was nutritional in four patients younger than 3 years of age and resulted from menstrual blood loss in two teenage girls. The mean initial hemoglobin was 2.5 g/dL (range 1.6-4.7) and the mean initial platelet count was 64 x 109/L (range 11-102). Bone marrow examinations were performed in three patients and showed increased numbers of megakaryocytes. After treatment with therapeutic doses of oral iron, all the patients showed rapid increases in their platelet counts. CONCLUSIONS: These observations validate and extend previous reports of an association between severe iron deficiency and thrombocytopenia. The increased numbers of megakaryocytes and the extremely rapid increase in platelet counts after initiation of iron therapy suggest an essential role for iron in a late stage of thrombopoiesis.
Administration, Oral↗