Validation of the MIBG SIOPEN scoring method in two independent high-risk neuroblastoma trials.

Konference: 2014 50th ASCO Annual Meeting - účast ČR

Kategorie: Nádory dětského a adolescentního věku; zhoubné nádory mozku a CNS

Téma: Postery

Číslo abstraktu: 10029

Autoři: MD Ariane Boubaker; Ulrike Poetschger, M.Sc.; M.D. Bieke Lambert; M.D. Maria Rita Castellani; M.D. Zvi Bar-Sever; Aurore Oudoux; Prof. M.D. Anna Kamińska; MUDr. Katarína Táborská; M.D. Lorenzo Biassoni; M.D. Susan G. Kreissman; M.D. Gregory A. Yanik; Prof. M.D. Arlene Naranjo, Ph.D.; M.D. Marguerite T. Parisi; M.D. Barry L. Shulkin; M.D. Katherine K. Matthay; M.D. Julie R. Park; Dr. Dominique Valteau Couanet; M.D. Ruth L Ladenstein, MBA


Background: Harmonised evaluation standards of MIBG scintigraphy for (re)staging of neuroblastoma (NB) are an international aim. In the HR-NBL1/SIOPEN trial population a SIOPEN score (SISCO) >3 was associated with a significantly poorer event free survival (EFS) on pre and post induction mIBG. This analysis validates the SISCO prognostic value in the independent dataset (DS) of the Children's Oncology Group (COG) protocol A3973. 

Methods: SIOPEN scoring evaluates mIBG uptake over 12 skeletal regions (scored 0-6/region, maximum of 72), not considering extra-osseous disease nor the primary site. MIBG scans from mIBG-avid stage 4 NB pts in 2 collaborative trials were reviewed and judged evaluable by the SIOPEN Nuclear Medicine review committee: the COG-A3973 (DSA; n=216) and SIOPEN HR-NBL1 trial (DSB; n=343). Predefined categories from DSB were used with a SISCO of 0, 1-3, 4-17 and ≥18. In addition, the YOUDEN index was used to identify single cut points (CPY). The median follow-up time was 7.1 and 5.5y, respectively. 

Results: Both DS showed a significantly superior EFS with a SISCO ≤3 at diagnosis [5-yr EFS in DSA: 51%±7% vs 34%±4%, p= 0.047 and in DSB 47%±7% vs 26%±3%, p=0.007]. However, the YOUDEN index yielded different results at diagnosis with a CPY of 40 in DSA and 10 in DSB and failed to identify a consistent poor prognosis group. A post induction SISCO of ≤3 also revealed a significant superior outcome [5-yr EFS in DSA: 43%±5% vs 16%±6%, p=0.004 and in DSB 36%±4% vs 14%±4%, p< 0.001]. The post induction CPY was 3 in DSB (in line with the DSB stratification criterion of ≤3 mIBG positive spots to proceed to high dose chemotherapy) and 0 in DSA. Pts with a SISCO of 0 post induction have the best outcome in both DS. In MYCN amplified pts, the pre and post-induction SISCO of ≤3 showed a significant impact in both groups, whilst in MYCN non-amplified pts this effect is only seen post induction. A SISCO ≤3 has independent statistical significance in Cox models including age and MYCN. 

Conclusions: A SIOPEN score ≥ 3 of mIBG scans carries relevant prognostic significance for the management of patients with high-risk NB at diagnosis and at the end of induction chemotherapy in the HRNBl1/SIOPEN trial and was confirmed in the independent COG A3973 data set. Clinical trial information: NCT01704716.

J Clin Oncol 32:5s, 2014 (suppl; abstr 10029)

Datum přednesení příspěvku: 2. 6. 2014