Cancer Res Treat.  2016 Oct;48(4):1408-1419. 10.4143/crt.2015.347.

Tandem High-Dose Chemotherapy and Autologous Stem Cell Transplantation for Atypical Teratoid/Rhabdoid Tumor

• Sung KW
• Lim DH
• Yi ES
• Choi YB
• Lee JW
• Yoo KH
• Koo HH
• Kim JH
• Suh YL
• Joung YS
• Shin HJ

• KMID: 2356243
• DOI: http://doi.org/10.4143/crt.2015.347

Abstract

PURPOSE
We prospectively evaluated the effectiveness of tandem high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) in improving the survival of patients with atypical teratoid/rhabdoid tumors while reducing the risks of late adverse effects from radiotherapy (RT).
MATERIALS AND METHODS
For young children (< 3 years old), tandem HDCT/auto-SCT was administered after six cycles of induction chemotherapy. RT was deferred until after 3 years of age unless the tumor showed relapse or progression. For older patients (> 3 years old), RT including reduced-dose craniospinal RT (23.4 or 30.6 Gy) was administered either after two cycles of induction chemotherapy or after surgery, and tandem HDCT/auto-SCT was administered after six cycles of induction chemotherapy.
RESULTS
A total of 13 patients (five young and eight older) were enrolled from November 2004 to June 2012. Eight patients, including all five young patients, had metastatic disease at diagnosis. Six patients (four young and two older) experienced progression before initiation of RT, and seven were able to proceed to HDCT/auto-SCT without progression during induction treatment. Three of six patients who experienced progression during induction treatment underwent HDCT/auto-SCT as salvage treatment. All five young patients died from disease progression. However, four of the eight older patients remain progression-freewith a median follow-up period of 64 months (range, 39 to 108 months). Treatment-related late toxicities were acceptable.
CONCLUSION
The required dose of craniospinal RT might be reduced in older patients if the intensity of chemotherapy is increased. However, early administration of RT should be considered to prevent early progression in young patients.

Keyword

Brain neoplasms; Chemotherapy; Hematopoietic stem cell transplantation; Radiotherapy

MeSH Terms

Brain Neoplasms
Child
Diagnosis
Disease Progression
Drug Therapy*
Follow-Up Studies
Hematopoietic Stem Cell Transplantation
Humans
Induction Chemotherapy
Prospective Studies
Radiotherapy
Recurrence
Salvage Therapy
Stem Cell Transplantation*
Stem Cells*

Figure

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  Fig. 1. Treatment scheme. For young children, six cycles of induction chemotherapy were administered prior to HDCT/auto-SCT, consisting of alternating CECV and CEIV regimens. RT was either not administered or was deferred until after 3 years of age if the patient achieved complete response after tandem HDCT/auto-SCT. Use of RT was determined according to the tumor status at the time of diagnosis. For older children, two cycles of pre-RT and four cycles of post-RT chemotherapy were administered during the early study period. During the late study period when RT was administered after surgery, six cycles of post-RT chemotherapy were administered. The regimens for the first and second HDCT/auto-SCT were CTE and CM, respectively. HDCT/auto-SCT, high-dose chemotherapy and autologous stem cell transplantation; CECV, carboplatin+etoposide+ cyclophosphamide+vincristine; CEIV, carboplatin+etoposide+ifosfamide+vincristine; RT, radiotherapy; CSRT, craniospinal RT; L-RT, local RT; Dx, diagnosis; CTE, carboplatin+thiotepa+etoposide; CM, cyclophosphamide+melphalan.

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  Fig. 2. Although all five young patients experienced relapse/progression and died from disease progression, four of the eight older patients remained event free at a median follow-up period of 64 months (range, 39 to 108 months) from diagnosis. For all patients, the 5-year event-free survival (EFS) and overall survival (OS) rates were 38.5±13.5% and 34.6±14.4%, respectively.

Reference

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