High-dose melphalan with autologous stem-cell rescue in metastatic rhabdomyosarcoma

Carli, M Colombatti, R Oberlin, O Stevens, M Masiero, L Frascella, E Koscielniak, E Treuner, J Pinkerton, CR

M. Carli et al., High-dose melphalan with autologous stem-cell rescue in metastatic rhabdomyosarcoma, J CL ONCOL, 17(9), 1999, pp. 2796-2803

33

INGLESE

Article

Oncology,"Onconogenesis & Cancer Research

JOURNAL OF CLINICAL ONCOLOGY

0732-183X → ACNP

17

9

1999

2796 - 2803

ISI

0732-183X(199909)17:9<2796:HMWASR>2.0.ZU;2-U

Purpose: The European Collaborative MMT4-91 trial was conducted as a prospe ctive nonrandomized study ta evaluate the potential benefit of high-dose me lphalan as consolidation of first complete remission in children with stage IV rhabdomyosarcoma, Patients and Methods: Fifty-two patients in complete remission after six co urses of chemotherapy received "megatherapy": 42 received melphalan alone, whereas 10 received melphalan in combination with etoposide, carboplatin/et oposide, or thiotepa/busuifan and etoposide, The outcome of this group of p atients was compared with that observed in 44 patients who were also in com plete remission after six courses of identical chemotherapy (plus surgery o r radiotherapy) but went on to receive a total of up to 12 courses of conve ntional chemotherapy (four cycles). No differences were found between the t wo groups regarding clinical characteristics, chemotherapy received before complete remission, or response to chemotherapy, In particular, there war n o significant difference between the groups for site of primary tumor, hist ologic subtype, age at presentation, presence of bone or bone marrow metast ases, or number of metastases. Results: The 3-year event-free survival (EFS) and overall survival (OS) Kit es were 29.7% and 40%, respectively, far those receiving high-dose melphala n or other multiagent high-dose regimens and 19.2% and 27.7%, respectively for those receiving standard chemotherapy. The difference was not statistic ally significant (P = .3 and P = .2 for EFS and OS, respectively), There wa s a significant prolongation in the time from the last day of high-dose che motherapy or the end of chemotherapy cycle 4 to the time of relapse in thos e receiving megatherapy (168 days far patients receiving megatherapy v 104 days far those receiving standard therapy; P =,05), Conclusion: The addition of a high-dose alkylating agent to consolidation t herapy may have prolonged progression-free survival in this poor-risk patie nt group, but it did not significantly improve the ultimate outcome.