Comment on: Consolidation treatment for high risk solid tumors in children with myeloablative chemotherapy and autologous hematopoietic progenitor stem cell transplantation (original) (raw)
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Bone Marrow Transplantation, 2013
Increasing treatment intensity has improved outcomes for children with neuroblastoma. We performed a pilot study in the Children's Oncology Group (COG) to assess feasibility and toxicity of a tandem myeloablative regimen without total body irradiation (TBI) supported by autologous CD34 selected peripheral blood stem cells. Forty-one patients with high-risk neuroblastoma were enrolled; eight patients did not receive any myeloablative consolidation procedure, and seven received only one. Two patients out of 41 (4.9%) experienced transplant-related mortality. CD34 selection was discontinued after subjects were enrolled due to serious viral illness. From the time Address reprint requests to: of study enrollment, the overall 3-year event-free survival (EFS) and overall survival (OS) were 44.8±9.6% and 59.2±9.2% (N=41). These results demonstrate that tandem transplantation in the cooperative group setting is feasible and support a randomized comparison of single versus tandem myeloablative consolidation with PBSC support for high-risk neuroblastoma.
Bone Marrow Transplantation, 1997
early recurrence of the primary tumor. The overall longterm survival rate is only 20-40%. 1,2 A number of approaches have been tried to improve the Between January 1993 and December 1996, 21 children with advanced solid tumors were entered in a dose-esca-result of HDCT. These approaches included increasing the number of agents in the myeloablative regimen and 'double lating study of high-dose sequential chemotherapy followed by autologous stem cell transplantation. The diag-grafting'-utilizing a second course of HDCT with an HSC infusion. Although preliminary data suggested improve-noses included neuroblastoma (NB) for 13 patients; Ewing's sarcoma (ES) for six patients and osteosarcoma ments in tumor response, significant obstacles were encountered. Extramedullary toxicities limit the dosage of each for two patients. Nine patients received therapy as consolidation for primary metastatic disease, and 12 drug in a multi-agent myeloablative regimen, 3 while cumulative toxicities and incomplete hematologic recovery patients had had previous relapses. Treatment consisted of CY given i.v. at a dose of 7 g/m 2 on day 1, followed between courses lead to a delay in treatment in a doublegrafting regimen. 4,5 Delaying treatment may result in tumor by G-CSF until myeloid recovery. After 3 weeks of rest, all patients were given thiotepa i.v. on days 22-24. The progression in high-risk patients and good responders may elect not to receive a second course of HDCT. An increase total dose of thiotepa was 450 mg/m 2 in three patients, 600 mg/m 2 in six patients, and 750 mg/m 2 in 12 patients. in treatment-related mortality has also been reported. 5,6 With these factors in mind, we developed a HDCT proto-Melphalan was given i.v. at a dose of 180 mg/m 2 i.v. on day 27 followed by stem cell infusion on day 28. Major col designed to deliver maximum or near-maximum tolerated doses in rapid sequence. The first course consisted of toxic reactions included stomatitis, esophagitis, diarrhea and dermatitis. Three patients died of treatment-related high-dose CY, which regularly produces severe myelosuppression followed by a predictable recovery within complications. Twelve patients have had a relapse. Six patients (five with NB and one with ES) are alive in 3 weeks; this was followed by a second course of bialkylator HDCT and autologous HSC infusion. We report the continuous remission 5-50 months (median 36) after transplantation. The results of this study show that it is results of a phase I-II dose-escalation study utilizing this approach. feasible to administer high-dose sequential chemotherapy to children with advanced solid tumors.
Pediatric Blood & Cancer, 2008
BackgroundIncreasing numbers of children with advanced neuroblastoma are achieving cure. We describe the clinical late effects specific to survivors of stage IV neuroblastoma all similarly treated using tandem autologous peripheral blood stem cell rescue with TBI.Increasing numbers of children with advanced neuroblastoma are achieving cure. We describe the clinical late effects specific to survivors of stage IV neuroblastoma all similarly treated using tandem autologous peripheral blood stem cell rescue with TBI.MethodThe medical records of 35 neuroblastoma patients treated at CHOP between 1997 and 2001 were examined. Eighteen of the 35 patients died of progressive disease, and 4 were lost to follow-up. Thirteen patients continue to follow-up in our Multidisciplinary Cancer Survivorship Clinic where they are evaluated and monitored by a consistent group of subspecialists that evaluate long-term sequelae. Data on treatment exposures including TBI and treatment related sequelae identified by clinician assessment and/or diagnostic testing were collected.The medical records of 35 neuroblastoma patients treated at CHOP between 1997 and 2001 were examined. Eighteen of the 35 patients died of progressive disease, and 4 were lost to follow-up. Thirteen patients continue to follow-up in our Multidisciplinary Cancer Survivorship Clinic where they are evaluated and monitored by a consistent group of subspecialists that evaluate long-term sequelae. Data on treatment exposures including TBI and treatment related sequelae identified by clinician assessment and/or diagnostic testing were collected.ResultsResults indicate late effects were present in all 13 subjects, 12 of whom suffered from multiple negative sequelae, including issues with growth hormone deficiency, dental problems, osteochondromas and hearing deficiencies, among others, most at higher rates than reported previously.Results indicate late effects were present in all 13 subjects, 12 of whom suffered from multiple negative sequelae, including issues with growth hormone deficiency, dental problems, osteochondromas and hearing deficiencies, among others, most at higher rates than reported previously.ConclusionsThe findings in this small cohort indicate the need for future prospective studies of this intensive pediatric cancer treatment, and underscore the importance of medical intervention and long-term monitoring of these at-risk subjects to increase overall quality-of-life. Pediatr Blood Cancer 2008;51:679–683. © 2008 Wiley-Liss, Inc.The findings in this small cohort indicate the need for future prospective studies of this intensive pediatric cancer treatment, and underscore the importance of medical intervention and long-term monitoring of these at-risk subjects to increase overall quality-of-life. Pediatr Blood Cancer 2008;51:679–683. © 2008 Wiley-Liss, Inc.
Bone Marrow Transplantation, 1997
than 20% have long-term survival. 1,2 Because chemotherapy dose-intensity has been shown to correlate strongly with response and progression-free survival in metastatic A single institutional pilot study was conducted in which 12 poor-risk neuroblastoma (NB) patients were uni-NB, 3 many centers are currently treating poor-risk patients with consolidation regimens that include myeloablative formly treated with multi-agent induction chemotherapy followed by myeloablative consolidation chemo-chemotherapy or chemoradiotherapy followed by allogeneic bone marrow transplant (BMT) or autologous BMT. therapy and unpurged peripheral blood stem cell (PBSC) rescue. In addition to using standard criteria While the efficacy of myeloablative vs nonmyeloablative consolidation therapy has not been determined, some of the for evaluating response to induction chemotherapy, tumor cell contamination of the peripheral blood and/or best long-term survival rates have been reported with highdose myeloablative chemotherapy with or without total-bone marrow was analyzed in seven patients by immunocytology using a panel of five anti-NB mono-body irradiation, and bone marrow rescue. 2,4-8 However, the ability to provide bone marrow stem cells for patient clonal antibodies. Seven patients had morphologic evidence of bone marrow disease at the time of diagnosis, rescue following intensive therapy can be limited by marrow contamination with tumor cells, and by the lack of and two additional patients had tumor cells detected in bone marrow samples by immunocytology prior to the adequate marrow reserves to harvest sufficient numbers of stem cells. second cycle of chemotherapy. After three cycles of chemotherapy, two of the 12 patients continued to have Rescue with peripheral blood stem cells (PBSCs) has several advantages over autologous bone marrow. The pro-evidence of bone marrow disease. Samples from 29 PBSC harvests collected from nine patients were also genitor cell collection can be performed on an outpatient basis without general anesthesia, and in general PBSCs can analyzed for the presence of contaminating tumor cells by immunocytology. In each case, the stem cells were be successfully harvested in patients who do not have adequate marrow reserves. 9 In addition, rescue with PBSCs found to be free of tumor. Eleven of the 12 patients underwent myeloablative therapy and PBSC rescue; results in a significantly reduced cytopenic period after myeloablative therapy. 9-11 Further, studies in adults have five patients remain alive without disease progression, 28+ to 53+ months from diagnosis, and six patients have indicated that the peripheral blood is enriched with stem cells during the recovery phase following chemotherapy, developed recurrent disease. We conclude that PBSCs can be successfully harvested from children with NB, and with proper timing and growth factor support, higher yields of stem cells can be obtained from peripheral stem and used for hematopoietic reconstitution following myeloablative chemotherapy. However, more effective cell harvests than from conventional bone marrow harvests. 9,12 Although the experience with PBSC harvest and therapy for poor-risk NB patients is still urgently needed.
2021
Objectives: The Primary aim of this study is to assess the outcome of children with solid and CNS tumors who underwent HDCT and ASCT at King Fahad Specialist Hospital in Dammam, secondarily we aim to compare our local results with national and international outcomes, Identify the toxicities, and complications of this modality of treatment, and identify the outcome in term of overall survival and relapse and mortality rates. Design: This is a retrospective cross-sectional study of all the pediatric patients below sixteen-year-old who diagnosed with solid tumor or central nervous system tumor and treated by high dose chemotherapy and autologous stem cell transplantation in king Fahd specialist hospital in Dammam, pediatric hematology/ oncology department between 1st November 2011 and 29th February 2020, our study included 33 cases. Setting: It is a single center study at King Fahad Specialist Hospital in Dammam which is 400 beds tertiary referral hospital with 27 beds pediatric oncolo...