Prolonged 14-day continuous infusion of high-dose ifosfamide for patients with relapsed and refractory high-grade osteosarcoma: a retrospective multicentre cohort study.


Journal

BMC cancer
ISSN: 1471-2407
Titre abrégé: BMC Cancer
Pays: England
ID NLM: 100967800

Informations de publication

Date de publication:
19 Jun 2024
Historique:
received: 10 02 2024
accepted: 10 06 2024
medline: 20 6 2024
pubmed: 20 6 2024
entrez: 19 6 2024
Statut: epublish

Résumé

The prognosis of patients with Relapsed/Refractory Osteosarcoma (R/R OS) remains dismal without an agreement on systemic therapy. The use of High-Dose Ifosfamide (14 g/sqm) with an external pump in outpatient setting (14-IFO) in R/R OS patients is limited. This study represents the first retrospective cohort analysis focused on evaluating the activity and toxicity of 14-IFO in this setting. The study investigated 14-IFO activity, in terms of tumour response according to RECIST 1.1 criteria, as well as survival rates and toxicity, according to CTCAE v.5. The trial enrolled 26 patients with R/R OS. The Overall Response Rate (ORR) and Disease Control Rate (DCR) obtained was 23% and 57.5%, respectively. Patients with relapsed OS showed a higher ORR (45%) and DCR (82%) compared to refractory patients, irrespective of the number of prior treatment lines received. The achievement of disease control with 14-IFO administration enabled 27% of patients to undergo new local treatment. Four-month Progression-Free Survival (PFS) was 54% for all patients and 82% for the relapsed OS sub-group. Median Overall Survival (OSurv) was 13.7 months, with 1-year OSurv of 51% for all patients and 71% for relapsed patients. Age over 18 years and the presence of refractory disease were identified as negative prognostic factors for this patient cohort. A total of 101 cycles were evaluated for toxic assessment, demonstrating a tolerable profile without grade 3-4 non-haematological toxicities. 14-IFO should be considered a viable treatment option for R/R OS, particularly due to its well tolerated toxicity profile and the potential for home-administration, which can improve patient quality of life without compromising efficacy.

Sections du résumé

BACKGROUND BACKGROUND
The prognosis of patients with Relapsed/Refractory Osteosarcoma (R/R OS) remains dismal without an agreement on systemic therapy. The use of High-Dose Ifosfamide (14 g/sqm) with an external pump in outpatient setting (14-IFO) in R/R OS patients is limited. This study represents the first retrospective cohort analysis focused on evaluating the activity and toxicity of 14-IFO in this setting.
PATIENTS AND METHODS METHODS
The study investigated 14-IFO activity, in terms of tumour response according to RECIST 1.1 criteria, as well as survival rates and toxicity, according to CTCAE v.5.
RESULTS RESULTS
The trial enrolled 26 patients with R/R OS. The Overall Response Rate (ORR) and Disease Control Rate (DCR) obtained was 23% and 57.5%, respectively. Patients with relapsed OS showed a higher ORR (45%) and DCR (82%) compared to refractory patients, irrespective of the number of prior treatment lines received. The achievement of disease control with 14-IFO administration enabled 27% of patients to undergo new local treatment. Four-month Progression-Free Survival (PFS) was 54% for all patients and 82% for the relapsed OS sub-group. Median Overall Survival (OSurv) was 13.7 months, with 1-year OSurv of 51% for all patients and 71% for relapsed patients. Age over 18 years and the presence of refractory disease were identified as negative prognostic factors for this patient cohort. A total of 101 cycles were evaluated for toxic assessment, demonstrating a tolerable profile without grade 3-4 non-haematological toxicities.
CONCLUSIONS CONCLUSIONS
14-IFO should be considered a viable treatment option for R/R OS, particularly due to its well tolerated toxicity profile and the potential for home-administration, which can improve patient quality of life without compromising efficacy.

Identifiants

pubmed: 38898388
doi: 10.1186/s12885-024-12498-x
pii: 10.1186/s12885-024-12498-x
doi:

Substances chimiques

Ifosfamide UM20QQM95Y
Antineoplastic Agents, Alkylating 0

Types de publication

Journal Article Multicenter Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

747

Informations de copyright

© 2024. The Author(s).

Références

Strauss SJ, Frezza AM, Abecassis N, Bajpai J, Bauer S, Biagini R, et al. Bone sarcomas: ESMO–EURACAN–GENTURIS–ERN PaedCan Clinical Practice Guideline for diagnosis, treatment and follow-up. Ann Oncol. 2021;32(12):1520–36.
doi: 10.1016/j.annonc.2021.08.1995 pubmed: 34500044
van Ewijk R, Herold N, Baecklund F, Baumhoer D, Boye K, Gaspar N, et al. European standard clinical practice recommendations for children and adolescents with primary and recurrent osteosarcoma. EJC Pediatr Oncol. 2023;2:100029.
doi: 10.1016/j.ejcped.2023.100029
Marina NM, Smeland S, Bielack SS, Bernstein M, Jovic G, Krailo MD, et al. Comparison of MAPIE versus MAP in patients with a poor response to preoperative chemotherapy for newly diagnosed high-grade osteosarcoma (EURAMOS-1): an open-label, international, randomised controlled trial. Lancet Oncol. 2016;17(10):1396–408.
doi: 10.1016/S1470-2045(16)30214-5 pubmed: 27569442 pmcid: 5052459
Bielack SS, Smeland S, Whelan JS, Marina N, Jovic G, Hook JM, et al. Methotrexate, Doxorubicin, and cisplatin (MAP) plus maintenance Pegylated Interferon Alfa-2b versus MAP alone in patients with Resectable High-Grade Osteosarcoma and good histologic response to preoperative MAP: first results of the EURAMOS-1 Good Response Randomized Controlled Trial. JCO. 2015;33(20):2279–87.
doi: 10.1200/JCO.2014.60.0734
Gaspar N, Occean BV, Pacquement H, Bompas E, Bouvier C, Brisse HJ, et al. Results of methotrexate-etoposide-ifosfamide based regimen (M-EI) in osteosarcoma patients included in the French OS2006/sarcome-09 study. Eur J Cancer. 2018;88:57–66.
doi: 10.1016/j.ejca.2017.09.036 pubmed: 29190507
Palmerini E, Meazza C, Tamburini A, Bisogno G, Ferraresi V, Asaftei SD, et al. Phase 2 study for nonmetastatic extremity high-grade osteosarcoma in pediatric and adolescent and young adult patients with a risk‐adapted strategy based on ABCB1/P‐glycoprotein expression: an Italian Sarcoma Group trial (ISG/OS‐2). Cancer. 2022;128(10):1958–66.
doi: 10.1002/cncr.34131 pubmed: 35201621
Jafari F, Javdansirat S, Sanaie S, Naseri A, Shamekh A, Rostamzadeh D, et al. Osteosarcoma: a comprehensive review of management and treatment strategies. Annals Diagn Pathol. 2020;49:151654.
doi: 10.1016/j.anndiagpath.2020.151654
Lagmay JP, Krailo MD, Dang H, Kim A, Hawkins DS, Beaty O, et al. Outcome of patients with recurrent Osteosarcoma enrolled in seven phase II trials through children’s Cancer Group, Pediatric Oncology Group, and Children’s Oncology Group: learning from the past to Move Forward. JCO. 2016;34(25):3031–8.
doi: 10.1200/JCO.2015.65.5381
Kempf-Bielack B, Bielack SS, Jürgens H, Branscheid D, Berdel WE, Exner GU, et al. Osteosarcoma Relapse after Combined Modality Therapy: an analysis of unselected patients in the Cooperative Osteosarcoma Study Group (COSS). JCO. 2005;23(3):559–68.
doi: 10.1200/JCO.2005.04.063
Ferrari S, Briccoli A, Mercuri M, Bertoni F, Picci P, Tienghi A, et al. Postrelapse Survival in Osteosarcoma of the extremities: prognostic factors for long-term survival. JCO. 2003;21(4):710–5.
doi: 10.1200/JCO.2003.03.141
Tirtei E, Asaftei SD, Manicone R, Cesari M, Paioli A, Rocca M, et al. Survival after second and subsequent recurrences in osteosarcoma: a retrospective multicenter analysis. Tumori. 2018;104(3):202–6.
doi: 10.1177/0300891617753257 pubmed: 30086698
Palmerini E, Setola E, Grignani G, D’Ambrosio L, Comandone A, Righi A, et al. High dose Ifosfamide in Relapsed and Unresectable High-Grade Osteosarcoma patients: a Retrospective Series. Cells. 2020;9(11):2389.
doi: 10.3390/cells9112389 pubmed: 33142760 pmcid: 7692098
Chou AJ, Merola PR, Wexler LH, Gorlick RG, Vyas YM, Healey JH, et al. Treatment of osteosarcoma at first recurrence after contemporary therapy: the Memorial Sloan-Kettering Cancer Center experience. Cancer. 2005;104(10):2214–21.
doi: 10.1002/cncr.21417 pubmed: 16206297
Verschoor AJ, Speetjens FM, Dijkstra PDS, Fiocco M, Sande MAJ, Bovée JVMG, et al. Single-center experience with Ifosfamide Monotherapy as Second-Line treatment of Recurrent/Metastatic osteosarcoma. Oncologist. 2020;25(4):e716–21.
doi: 10.1634/theoncologist.2019-0528 pubmed: 32297446
Harris MB, Cantor AB, Goorin AM, Shochat SJ, Ayala AG, Ferguson WS, et al. Treatment of osteosarcoma with ifosfamide: comparison of response in pediatric patients with recurrent disease versus patients previously untreated: a pediatric oncology group study. Med Pediatr Oncol. 1995;24(2):87–92.
doi: 10.1002/mpo.2950240205 pubmed: 7990769
Patel SR, Vadhan-Raj S, Papadopolous N, Plager C, Burgess MA, Hays C, et al. High-dose ifosfamide in bone and soft tissue sarcomas: results of phase II and pilot studies–dose-response and schedule dependence. JCO. 1997;15(6):2378–84.
doi: 10.1200/JCO.1997.15.6.2378
Berrak SG, Pearson M, Berberoğlu S. Ilhan Inci ErgüRhan, Jaffe N. High-dose ifosfamide in relapsed pediatric osteosarcoma: therapeutic effects and renal toxicity. Pediatr Blood Cancer. 2005;44(3):215–9.
Ferrari S, Zolezzi C, Cesari M, Fasano MC, Lamanna G, Bacci G. Prospective evaluation of high-dose ifosfamide-related nephrotoxicity in young adult patients with recurrent osteosarcoma previously treated with cisplatin, methotrexate and standard-dose ifosfamide. Anticancer Drugs. 1999;10(1):25–32.
doi: 10.1097/00001813-199901000-00004 pubmed: 10194544
Carter TJ, Milic M, McDerra J, McTiernan A, Ahmed M, Karavasilis V, et al. Continuous 14 day Infusional Ifosfamide for Management of Soft-tissue and bone sarcoma: a single Centre Retrospective Cohort Analysis. Cancers. 2020;12(11):3408.
doi: 10.3390/cancers12113408 pubmed: 33212978 pmcid: 7698576
Meazza C, Casanova M, Luksch R, Podda M, Favini F, Cefalo G, et al. Prolonged 14-day continuous infusion of high-dose ifosfamide with an external portable pump: feasibility and efficacy in refractory pediatric sarcoma. Pediatr Blood Cancer. 2010;55(4):617–20.
doi: 10.1002/pbc.22596 pubmed: 20589638
Cerny T, Castiglione M, Brunner K, Küpfer A, Martinelli G, Lind M. Ifosfamide by continuous infusion to prevent encephalopathy. Lancet. 1990;335(8682):175.
doi: 10.1016/0140-6736(90)90053-8 pubmed: 1967471
Boddy AV, Yule SM, Wyllie R, Price L, Pearson ADJ, Idle JR. Comparison of continuous infusion and bolus administration of ifosfamide in children. Eur J Cancer. 1995;31(5):785–90.
doi: 10.1016/0959-8049(95)00090-6
Palmerini E, Jones RL, Marchesi E, Paioli A, Cesari M, Longhi A, et al. Gemcitabine and docetaxel in relapsed and unresectable high-grade osteosarcoma and spindle cell sarcoma of bone. BMC Cancer. 2016;16(1):280.
doi: 10.1186/s12885-016-2312-3 pubmed: 27098543 pmcid: 4839113
Song BS, Seo J, Kim DH, Lim JS, Yoo JY, Lee JA. Gemcitabine and docetaxel for the treatment of children and adolescents with recurrent or refractory osteosarcoma: Korea Cancer Center Hospital experience. Pediatr Blood Cancer. 2014;61(8):1376–81.
doi: 10.1002/pbc.25035 pubmed: 24692087
Navid F, Willert JR, McCarville MB, Furman W, Watkins A, Roberts W, et al. Combination of gemcitabine and docetaxel in the treatment of children and young adults with refractory bone sarcoma. Cancer. 2008;113(2):419–25.
doi: 10.1002/cncr.23586 pubmed: 18484657
Fox E, Patel S, Wathen JK, Schuetze S, Chawla S, Harmon D, et al. Phase II study of Sequential Gemcitabine followed by Docetaxel for Recurrent Ewing Sarcoma, Osteosarcoma, or unresectable or locally recurrent Chondrosarcoma: results of Sarcoma Alliance for Research through collaboration Study 003. Oncologist. 2012;17(3):321–e329.
doi: 10.1634/theoncologist.2010-0265 pubmed: 22363068 pmcid: 3316916
Berger M, Grignani G, Ferrari S, Biasin E, Brach del Prever A, Aliberti S, et al. Phase 2 trial of two courses of cyclophosphamide and etoposide for relapsed high-risk osteosarcoma patients. Cancer. 2009;115(13):2980–7.
doi: 10.1002/cncr.24368 pubmed: 19452540
Rodríguez-Galindo C, Daw NC, Kaste SC, Meyer WH, Dome JS, Pappo AS, et al. Treatment of refractory osteosarcoma with fractionated Cyclophosphamide and Etoposide. J Pediatr Hematol Oncol. 2002;24(4):250–5.
doi: 10.1097/00043426-200205000-00006 pubmed: 11972091
Miser JS, Kinsella TJ, Triche TJ, Tsokos M, Jarosinski P, Forquer R, et al. Ifosfamide with mesna uroprotection and etoposide: an effective regimen in the treatment of recurrent sarcomas and other tumors of children and young adults. JCO. 1987;5(8):1191–8.
doi: 10.1200/JCO.1987.5.8.1191
Kung FH, Pratt CB, Vega RA, Jaffe N, Strother D, Schwenn M, et al. Ifosfamide/etoposide combination in the treatment of recurrent malignant solid tumors of childhood. A pediatric oncology group phase II study. Cancer. 1993;71(5):1898–903.
doi: 10.1002/1097-0142(19930301)71:5<1898::AID-CNCR2820710529>3.0.CO;2-Q pubmed: 8448755
Coriat R, Mir O, Camps S, Ropert S, Billemont B, Leconte M, et al. Ambulatory administration of 5-day infusion ifosfamide + mesna: a pilot study in sarcoma patients. Cancer Chemother Pharmacol. 2010;65(3):491–5.
doi: 10.1007/s00280-009-1054-1 pubmed: 19588140
Loeffler TM, Weber FW, Hausamen TU. Ambulatory high-dose 5-day continuous-infusion ifosfamide combination chemotherapy in advanced solid tumors: a feasibility study. J Cancer Res Clin Oncol. 1991;117(S4):S125–8.
doi: 10.1007/BF01613216 pubmed: 1795000
Schwartz LH, Seymour L, Litière S, Ford R, Gwyther S, Mandrekar S, et al. RECIST 1.1 – standardisation and disease-specific adaptations: perspectives from the RECIST Working Group. Eur J Cancer. 2016;62:138–45.
doi: 10.1016/j.ejca.2016.03.082 pubmed: 27237360 pmcid: 5737786
NCCN. Guidelines_Bone Sarcoma.pdf.
Wiener L, Zadeh S, Battles H, Baird K, Ballard E, Osherow J, et al. Allowing adolescents and young adults to Plan their end-of-Life Care. Pediatrics. 2012;130(5):897–905.
doi: 10.1542/peds.2012-0663 pubmed: 23045560 pmcid: 3483891
Di Cataldo A, Astuto M, Rizzo G, Bertuna G, Russo G, Incorpora G. Neurotoxicity during ifosfamide treatment in children. Med Sci Monit. 2009;15(1):CS22–5.
pubmed: 19114973
Idle JR. Diren Beyoğlu,. Ifosfamide - History, efficacy, toxicity and encephalopathy. Pharmacol Ther. 2023;243:108–366.
doi: 10.1016/j.pharmthera.2023.108366
Von Hoff DD. There are no bad anticancer agents, only bad clinical trial designs–twenty-first Richard and Hinda Rosenthal Foundation Award lecture. Clin Cancer Res. 1998;4:1079–86.
Italiano A, Mir O, Mathoulin-Pelissier S, Penel N, Piperno-Neumann S, Bompas E, Chevreau C, Duffaud F, Entz-Werlé N, Saada E, Ray-Coquard I, Lervat C, Gaspar N, Marec-Berard P, Pacquement H, Wright J, Toulmonde M, Bessede A, Crombe A, Kind M, Bellera C, Blay JY. Cabozantinib in patients with advanced ewing sarcoma or osteosarcoma (CABONE): a multicentre, single-arm, phase 2 trial. Lancet Oncol. 2020;21(3):446–55.
doi: 10.1016/S1470-2045(19)30825-3 pubmed: 32078813 pmcid: 8763616

Auteurs

Elisa Tirtei (E)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.
Department of Public Health and Paediatrics, University of Turin, Turin, Italy.

Anna Campello (A)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy. anna.campello@unito.it.

Veronica Sciannameo (V)

Centre for Biostatistics, Epidemiology and Public Health, Department of Clinical and Biological Sciences, University of Turin, Regione Gonzole 10, Orbassano, 10043, Italy. veronica.sciannameo@unito.it.

Sebastian Dorin Asaftei (SD)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.

Cristina Meazza (C)

Paediatric Oncology Unit, Fondazione IRCCS, Istituto Nazionale dei Tumori, Milan, Italy.

Giovanna Sironi (G)

Paediatric Oncology Unit, Fondazione IRCCS, Istituto Nazionale dei Tumori, Milan, Italy.

Alessandra Longhi (A)

Osteoncology, Bone and Soft Tissue Sarcomas and Innovative Therapies, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.

Toni Ibrahim (T)

Osteoncology, Bone and Soft Tissue Sarcomas and Innovative Therapies, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.

Angela Tamburini (A)

Department of Paediatric Haematology-Oncology, Meyer Children's Hospital IRCCS, Florence, Italy.

Luca Coccoli (L)

Pediatric Oncology-Hematology Unit, Stem Cell Transplantation and EURACAN Hub Center Unit, S. Chiara Hospital, AOUP, Pisa, Italy.

Fanj Crocco (F)

Paediatrics Division, Department of Health Sciences, AOU Maggiore della Carità di Novara, Piemonte Orientale University, Novara, Italy.

Celeste Cagnazzo (C)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.

Elvira De Luna (E)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.

Paola Quarello (P)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.
Department of Public Health and Paediatrics, University of Turin, Turin, Italy.

Paola Berchialla (P)

Centre for Biostatistics, Epidemiology and Public Health, Department of Clinical and Biological Sciences, University of Turin, Regione Gonzole 10, Orbassano, 10043, Italy.

Franca Fagioli (F)

Paediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Regina Margherita Children's Hospital, Piazza Polonia 94, Turin, 10126, Italy.
Department of Public Health and Paediatrics, University of Turin, Turin, Italy.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH