Korean J Pediatr.  2018 Feb;61(2):53-58. 10.3345/kjp.2018.61.2.53.

Excellent treatment outcomes in children younger than 18 months with stage 4 MYCN nonamplified neuroblastoma

• Kim C
• Choi YB
• Lee JW
• Yoo KH
• Sung KW
• Koo HH

• KMID: 2452041
• DOI: http://doi.org/10.3345/kjp.2018.61.2.53

Abstract

PURPOSE
Although the prognosis is generally good in patients with intermediate-risk neuroblastoma, no consensus has been reached on the ideal treatment regimen. This study analyzed treatment outcomes and toxicities in patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma.
METHODS
We retrospectively analyzed 20 patients younger than 18 months newly diagnosed with stage 4 MYCN nonamplified neuroblastoma between January 2009 and December 2015. Patients received 9 cycles of chemotherapy and surgery, with or without local radiotherapy, followed by 12 cycles of differentiation therapy with 13-cis-retinoic acid. Chemotherapy consisted of alternating cycles of cisplatin, etoposide, doxorubicin, and cyclophosphamide (CEDC) and ifosfamide, carboplatin, and etoposide (ICE) regimens.
RESULTS
The most common primary tumor site was the abdomen (85%), and the most common metastatic sites were the lymph nodes (65%), followed by the bones (60%), liver (55%), skin (45%), and bone marrow (25%). At the end of induction therapy, 14 patients (70%) achieved complete response, with 1 achieving very good partial response, 4 achieving partial response, and 1 showing mixed response. Nine patients (45%) received local radiotherapy. At a median follow-up of 47 months (range, 17-91 months), none of these patients experienced relapse, progression, or secondary malignancy, or died. Three years after chemotherapy completion, none of the patients had experienced grade ≥3 late adverse effects.
CONCLUSION
Patients younger than 18 months with stage 4 MYCN nonamplified neuroblastoma showed excellent outcomes, without significant late adverse effects, when treated with alternating cycles of CEDC and ICE, followed by surgery and differentiation therapy.

Keyword

Neuroblastoma; Infant; Neoplasm metastasis; N-myc proto-oncogene protein

MeSH Terms

Abdomen
Bone Marrow
Carboplatin
Child*
Cisplatin
Consensus
Cyclophosphamide
Doxorubicin
Drug Therapy
Etoposide
Follow-Up Studies
Humans
Ice
Ifosfamide
Infant
Isotretinoin
Liver
Lymph Nodes
Neoplasm Metastasis
Neuroblastoma*
Prognosis
Radiotherapy
Recurrence
Retrospective Studies
Skin
Carboplatin
Cisplatin
Cyclophosphamide
Doxorubicin
Etoposide
Ice
Ifosfamide
Isotretinoin