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| Name | Class |
|---|---|
| Ruijin Hospital | OTHER |
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Evaluate the impact of maintenance therapy with eflornithine on event-free survival and overall survival in high-risk neuroblastoma (NB) children after immunotherapy, and assess its safety.
The investigators plan to recruit 20 participants and divide them into Group A and Group B based on their clinical status. The inclusion criteria for Group A are: newly diagnosed high-risk neuroblastoma (NB) patients who have completed the standard treatment plan (including immunotherapy) and whose disease has reached complete response (CR) or very good partial response (VGPR). The inclusion criteria for Group B are: recurrent or refractory NB patients who have completed any treatment for recurrent disease, or who have achieved disease stability after any salvage or intensification therapy for primary refractory disease, with at least a partial response (PR) as assessed by CT or MRI and negative bone marrow aspiration. There are no restrictions regarding gender or geographic region. Participants must be under 18 years of age. Eligible subjects will be enrolled into the study after receiving the first oral dose of eflornithine, with a total treatment course of 2 years. Follow-up will continue for up to 3 years after completion of eflornithine therapy.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oral administration of eflornithine for the prevention of high-risk neuroblastoma | Drug | This study is a single arm clinical observational study and does not involve control interventions |
|
| Measure | Description | Time Frame |
|---|---|---|
| The event free survival rate after the completion of the first DFMO | The event free survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy. | 5 years |
| overall survival rate after the completion of the first DFMO | overall survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy. | 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events | This study will comprehensively evaluate the safety and tolerability of eflornithine. The primary assessments include the incidence and severity of all treatment-emergent adverse events (graded per NCI CTCAE v5.0) | 5 years |
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Inclusion Criteria:
Exclusion Criteria:
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Children with high-risk neuroblastoma
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| Name | Affiliation | Role |
|---|---|---|
| Jingbo Shao, PhD,chief physician | Shanghai Children's Hospital | Principal Investigator |
| Wen Su, PhD,associate chief physician | Ruijin Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ruijin Hospital Hainan Branch, affiliated with Shanghai Jiao Tong University School of Medicine and Children's Hospital of Shanghai, affiliated with Shanghai Jiao Tong University School of Medicine | Hainan | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38917371 | Result | Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25. | |
| 32697811 |
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| Result |
| Diccianni MB, Kempinska K, Gangoti JA, Yu AL, Sorkin LS. Anti-GD2 induced allodynia in rats can be reduced by pretreatment with DFMO. PLoS One. 2020 Jul 22;15(7):e0236115. doi: 10.1371/journal.pone.0236115. eCollection 2020. |
| 35605657 | Result | Salemi F, Alam W, Hassani MS, Hashemi SZ, Jafari AA, Mirmoeeni SMS, Arbab M, Mortazavizadeh SMR, Khan H. Neuroblastoma: Essential genetic pathways and current therapeutic options. Eur J Pharmacol. 2022 Jul 5;926:175030. doi: 10.1016/j.ejphar.2022.175030. Epub 2022 May 20. |
| 31094257 | Result | Jabbari P, Hanaei S, Rezaei N. State of the art in immunotherapy of neuroblastoma. Immunotherapy. 2019 Jun;11(9):831-850. doi: 10.2217/imt-2019-0018. Epub 2019 May 16. |
| 39062971 | Result | Pieniazek B, Cencelewicz K, Bzdziuch P, Mlynarczyk L, Lejman M, Zawitkowska J, Derwich K. Neuroblastoma-A Review of Combination Immunotherapy. Int J Mol Sci. 2024 Jul 15;25(14):7730. doi: 10.3390/ijms25147730. |
| 26304901 | Result | Pinto NR, Applebaum MA, Volchenboum SL, Matthay KK, London WB, Ambros PF, Nakagawara A, Berthold F, Schleiermacher G, Park JR, Valteau-Couanet D, Pearson AD, Cohn SL. Advances in Risk Classification and Treatment Strategies for Neuroblastoma. J Clin Oncol. 2015 Sep 20;33(27):3008-17. doi: 10.1200/JCO.2014.59.4648. Epub 2015 Aug 24. |
| 30305231 | Result | Newman EA, Abdessalam S, Aldrink JH, Austin M, Heaton TE, Bruny J, Ehrlich P, Dasgupta R, Baertschiger RM, Lautz TB, Rhee DS, Langham MR Jr, Malek MM, Meyers RL, Nathan JD, Weil BR, Polites S, Madonna MB; APSA Cancer committee. Update on neuroblastoma. J Pediatr Surg. 2019 Mar;54(3):383-389. doi: 10.1016/j.jpedsurg.2018.09.004. Epub 2018 Sep 19. |
| 30984908 | Result | Hubbard AK, Spector LG, Fortuna G, Marcotte EL, Poynter JN. Trends in International Incidence of Pediatric Cancers in Children Under 5 Years of Age: 1988-2012. JNCI Cancer Spectr. 2019 Mar;3(1):pkz007. doi: 10.1093/jncics/pkz007. Epub 2019 Apr 9. |