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Article: A phase I/II study of CY and topotecan in patients with high-risk malignancies undergoing autologous hematopoietic cell transplantation: The St Jude long-term follow-up

TitleA phase I/II study of CY and topotecan in patients with high-risk malignancies undergoing autologous hematopoietic cell transplantation: The St Jude long-term follow-up
Authors
KeywordsCY
sarcoma
neuroblastoma
autologous BMT
lymphoma
topotecan
Issue Date2012
Citation
Bone Marrow Transplantation, 2012, v. 47, n. 11, p. 1448-1454 How to Cite?
AbstractFifty-eight consecutive children with high-risk malignancies were treated with CY, and targeted topotecan followed by autologous hematopoietic cell transplantation (AHCT) in a phase I/II Institutional Review Board-approved study. Twelve participants enrolled in phase I; 5 received dose level 1 of topotecan 3 mg/m2 per day, with subsequent doses targeted to total systemic exposure of 100±20 ng h/mL and CY 750 mg/m2 per day. Seven participants received dose level 2. CY dose escalation to 1 g/m 2 per day was considered excessively toxic; one died from irreversible veno-occlusive disease and two experienced reversible hepatotoxicity. These adverse events halted further dose escalation. A total of 46 participants were enrolled in phase II; results are on the 51 participants who received therapy at dose level 1, the maximum tolerated dose. Diagnoses included neuroblastoma (26), sarcoma (9), lymphoma (8), brain tumors (5), Wilms (2) and retinoblastoma (1). Twenty participants (39.3%) were in CR1 at enrollment; median age was 5.1 years. Most common non-hematological grade IIIIV toxicity was gastrointestinal (n37). Neutrophil and platelet engraftment occurred at a median of 15 and 24 days, respectively. Twenty-six (51%) participants remain alive at a median of 6.4 years after AHCT. CY 3.75 g/m 2, and targeted topotecan followed by AHCT are feasible and produce acceptable toxicity in children with high-risk malignancies. © 2012 Macmillan Publishers Limited All rights reserved.
Persistent Identifierhttp://hdl.handle.net/10722/294492
ISSN
2023 Impact Factor: 4.5
2023 SCImago Journal Rankings: 1.318
PubMed Central ID
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorKasow, K. A.-
dc.contributor.authorStewart, C. F.-
dc.contributor.authorBarfield, R. C.-
dc.contributor.authorWright, N. L.-
dc.contributor.authorLi, C.-
dc.contributor.authorSrivastava, D. K.-
dc.contributor.authorLeung, W.-
dc.contributor.authorHorwitz, E. M.-
dc.contributor.authorBowman, L. C.-
dc.contributor.authorHandgretinger, R.-
dc.contributor.authorHale, G. A.-
dc.date.accessioned2020-12-03T08:22:52Z-
dc.date.available2020-12-03T08:22:52Z-
dc.date.issued2012-
dc.identifier.citationBone Marrow Transplantation, 2012, v. 47, n. 11, p. 1448-1454-
dc.identifier.issn0268-3369-
dc.identifier.urihttp://hdl.handle.net/10722/294492-
dc.description.abstractFifty-eight consecutive children with high-risk malignancies were treated with CY, and targeted topotecan followed by autologous hematopoietic cell transplantation (AHCT) in a phase I/II Institutional Review Board-approved study. Twelve participants enrolled in phase I; 5 received dose level 1 of topotecan 3 mg/m2 per day, with subsequent doses targeted to total systemic exposure of 100±20 ng h/mL and CY 750 mg/m2 per day. Seven participants received dose level 2. CY dose escalation to 1 g/m 2 per day was considered excessively toxic; one died from irreversible veno-occlusive disease and two experienced reversible hepatotoxicity. These adverse events halted further dose escalation. A total of 46 participants were enrolled in phase II; results are on the 51 participants who received therapy at dose level 1, the maximum tolerated dose. Diagnoses included neuroblastoma (26), sarcoma (9), lymphoma (8), brain tumors (5), Wilms (2) and retinoblastoma (1). Twenty participants (39.3%) were in CR1 at enrollment; median age was 5.1 years. Most common non-hematological grade IIIIV toxicity was gastrointestinal (n37). Neutrophil and platelet engraftment occurred at a median of 15 and 24 days, respectively. Twenty-six (51%) participants remain alive at a median of 6.4 years after AHCT. CY 3.75 g/m 2, and targeted topotecan followed by AHCT are feasible and produce acceptable toxicity in children with high-risk malignancies. © 2012 Macmillan Publishers Limited All rights reserved.-
dc.languageeng-
dc.relation.ispartofBone Marrow Transplantation-
dc.subjectCY-
dc.subjectsarcoma-
dc.subjectneuroblastoma-
dc.subjectautologous BMT-
dc.subjectlymphoma-
dc.subjecttopotecan-
dc.titleA phase I/II study of CY and topotecan in patients with high-risk malignancies undergoing autologous hematopoietic cell transplantation: The St Jude long-term follow-up-
dc.typeArticle-
dc.description.naturelink_to_OA_fulltext-
dc.identifier.doi10.1038/bmt.2012.51-
dc.identifier.pmid22426752-
dc.identifier.pmcidPMC4893814-
dc.identifier.scopuseid_2-s2.0-84869089853-
dc.identifier.volume47-
dc.identifier.issue11-
dc.identifier.spage1448-
dc.identifier.epage1454-
dc.identifier.eissn1476-5365-
dc.identifier.isiWOS:000310795500011-
dc.identifier.issnl0268-3369-

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