High‐dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high‐risk or relapsed medulloblastoma or supratentorial primitive neuroectodermal tumor

Sung, Ki Woong; Yoo, Keon Hee; Cho, Eun Joo; Koo, Hong Hoe; Lim, Do Hoon; Shin, Hyung Jin; Ahn, Seung Do; Shin, Young; Choi, Eun Seok; Ghim, Thad T.

doi:10.1002/pbc.21064

Cited by 68 publications

(59 citation statements)

References 28 publications

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“…26 High-dose chemotherapy with stem cell support may also be beneficial in PNET and medulloblastoma patients, albeit at the cost of significant acute toxicity. [27][28][29][30] Conventional multiagent chemotherapy was able to cure children with earlystage, completely resected newly diagnosed medulloblastoma, but was insufficient for treatment of metastatic or incompletely resected tumors. 31 Many patients in our study had adverse prognostic factors.…”

Section: Supratentorial and Disseminated Pnets Medulloblastomasmentioning

confidence: 99%

Intraarterial chemotherapy and osmotic blood‐brain barrier disruption for patients with embryonal and germ cell tumors of the central nervous system

Jahnke

Kraemer

Knight

et al. 2007

Cancer

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BACKGROUND. The rate of durable responses in embryonal and certain germ cell tumors of the central nervous system (CNS) is unsatisfactory. Intraarterial chemotherapy and osmotic blood‐brain barrier disruption (IA/BBBD) increases drug delivery to the CNS. METHODS. Data of patients treated with carboplatin or methotrexate‐based IA/BBBD on prospective phase 2 trials conducted at 3 centers were collected. Study outcomes included overall survival (OS), time to progression (TTP), and toxicity. RESULTS. Fifty‐four patients were treated. Twenty‐seven patients received IA/BBBD as salvage treatment. The median OS was 2.8 years for all patients, 2.5 years for supratentorial and disseminated primitive neuroectodermal tumors (PNETs, n = 29), 1.7 years for medulloblastomas (n = 12), and 5.4 years for germ cell tumors (n = 13). OS and TTP for all patients were better with a Karnofsky Performance Status ≥70% (P = .0013 and .0070) and IA/BBBD as first‐line treatment (P = .0059 and .029). In PNETs, OS was higher with pineal location (P = .045) and IA/BBBD as first‐line treatment (P = .0036), and TTP was improved with radiotherapy before IA/BBBD (P = .036) and IA/BBBD as first‐line treatment (P = .0079). Seventeen of 54 patients (31%) are alive, and 16 are alive at 4+ to 18+ years. Three survivors were not treated with radiotherapy and 4 were treated with focal radiotherapy only. The patients who were not irradiated did not develop dementia. CONCLUSIONS. Survival and toxicity data appear promising, considering the cohort's adverse prognostic profile. A plateau in survival curves suggests a cure for some patients. Long‐term survival may be achieved with focal or reduced‐dose radiotherapy in some IA/BBBD patients. Cancer 2008. © 2007 American Cancer Society.

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Section: Supratentorial and Disseminated Pnets Medulloblastomasmentioning

confidence: 99%

Intraarterial chemotherapy and osmotic blood‐brain barrier disruption for patients with embryonal and germ cell tumors of the central nervous system

Jahnke

Kraemer

Knight

et al. 2007

Cancer

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“…Recently, investigators have examined the efficacy of double or tripletandem HDCT/ASCR for further improvements in the outcome of patients with high-risk solid tumors. Sung et al 8,9 reported improved long-term survival using tandem HDCT/ASCR in patients with advanced neuroblastoma and high-risk brain tumors. Kletzel et al 10 also reported improved survival in a single-arm trial of triple-tandem HDCT/ASCR in patients with high-risk neuroblastoma.…”

Section: Introductionmentioning

confidence: 99%

“…[7][8][9][10] This treatment strategy is based on the hypothesis that a dose escalation might improve the survival of children with high-risk solid tumors. Recently, investigators have examined the efficacy of double or tripletandem HDCT/ASCR for further improvements in the outcome of patients with high-risk solid tumors.…”

Section: Introductionmentioning

confidence: 99%

Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma

et al. 2008

Bone Marrow Transplant

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“…[11][12][13] Additionally, several recent studies have suggested that dose-escalation using tandem HDCT/auto-SCT might further improve outcomes in the treatment of recurrent or high-risk brain tumors. 14,15 In the present study, we prospectively evaluated the feasibility and effectiveness of tandem HDCT/auto-SCT in children o3 years of age with malignant brain tumors. RT was either not given or deferred until 3 years of age if the patient achieved CR after tandem HDCT/auto-SCT.…”

Section: Introductionmentioning

confidence: 99%

Tandem high-dose chemotherapy and auto-SCT for malignant brain tumors in children under 3 years of age

et al. 2013

Bone Marrow Transplant

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In an effort to improve survival and reduce late adverse effects of radiation therapy (RT), 25 children o3 years of age with malignant brain tumors received tandem high-dose chemotherapy (HDCT) and auto-SCT following six cycles of induction chemotherapy. RT was either not given or deferred until 3 years of age if the patient was in CR after tandem HDCT/auto-SCT. Tumors relapsed or progressed in nine patients (five during induction treatment), and two of these patients survived after receiving salvage treatment, including RT. Two patients died due to toxicities during tandem HDCT/auto-SCT. A total of 16 patients survived to a median follow-up period of 52 months (range 18-96) from the time of diagnosis. Four of these patients did not receive RT, two received local RT (L-RT), three received craniospinal RT (CSRT), and seven received both L-RT and CSRT. The 5-year OS and EFS rates were 67.8 ± 9.4% and 55.5 ± 10.0%, respectively. Neuroendocrine and neurocognitive functions evaluated 3 years after tandem HDCT/auto-SCT were acceptable. Our results indicate that tandem HDCT/auto-SCT may improve survival in young children with malignant brain tumors with an acceptable level of risk of long-term toxicity.

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High‐dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high‐risk or relapsed medulloblastoma or supratentorial primitive neuroectodermal tumor

Abstract: High-dose chemotherapy may improve the survival of children with newly diagnosed high-risk MB/sPNET, and, to some extent, the survival of those with relapsed MB/sPNET. Further study is necessary to elucidate the efficacy of tandem double HDCT.

Cited by 68 publications

References 28 publications

Intraarterial chemotherapy and osmotic blood‐brain barrier disruption for patients with embryonal and germ cell tumors of the central nervous system

Intraarterial chemotherapy and osmotic blood‐brain barrier disruption for patients with embryonal and germ cell tumors of the central nervous system

Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma

Tandem high-dose chemotherapy and auto-SCT for malignant brain tumors in children under 3 years of age

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