Validation of the mIBG skeletal SIOPEN scoring method in two independent high-risk neuroblastoma populations: the SIOPEN/HR-NBL1 and COG-A3973 trials

被引：52

作者：

Ladenstein, Ruth ^{[1
,2
]}

Lambert, Bieke ^{[3
]}

Potschger, Ulrike ^{[1
]}

Castellani, Maria-Rita ^{[4
]}

Lewington, Valerie ^{[5
]}

Bar-Sever, Zvi ^{[6
]}

Oudoux, Aurore ^{[7
]}

Sliwinska, Anna ^{[8
]}

Taborska, Katerina ^{[9
]}

Biassoni, Lorenzo ^{[10
]}

Yanik, Gregory A. ^{[11
]}

Naranjo, Arlene ^{[12
]}

Parisi, Marguerite T. ^{[13
]}

Shulkin, Barry L. ^{[14
]}

Nadel, Helen ^{[15
]}

Gelfand, Michael J. ^{[16
]}

Matthay, Katherine K. ^{[17
]}

Park, Julie R. ^{[18
]}

Kreissman, Susan G. ^{[19
]}

Valteau-Couanet, Dominique ^{[20
]}

Boubaker, Ariane ^{[21
]}

机构：

[1] Childrens Canc Res Inst, Zimmermannpl 10, A-1090 Vienna, Austria

[2] Med Univ, Dept Paediat, Vienna, Austria

[3] Univ Ghent, Radiol & Nucl Med, Ghent, Belgium

[4] Fdn IRCCS Ist Nazl Tumori, Nucl Med Div, Milan, Italy

[5] Guys & St Thomas NHS Fdn Trust, London, England

[6] Schneider Childrens Med Ctr Israel, Petah Tiqwa, Israel

[7] Oscar Lambret Ctr, Dept Nucl Med Lille, Lille, France

[8] Childrens Mem Hlth Inst, Warsaw, Poland

[9] Motol Univ Hosp, Prague, Czech Republic

[10] Great Ormond St Hosp Children NHS Fdn Trust, London, England

[11] Univ Michigan, Med Ctr, Ann Arbor, MI USA

[12] Univ Florida, Childrens Oncol Grp, Stat & Data Ctr, Gainesville, FL USA

[13] Univ Washington, Sch Med, Dept Radiol, Seattle Childrens Hosp, Seattle, WA 98195 USA

[14] St Jude Childrens Res Hosp, Dept Diagnost Imaging, 332 N Lauderdale St, Memphis, TN 38105 USA

[15] BC Childrens Hosp, Dept Radiol, Vancouver, BC, Canada

[16] Cincinnati Childrens Hosp Med Ctr, Dept Radiol, Cincinnati, OH 45229 USA

[17] Univ Calif San Francisco, Sch Med, Dept Pediat, San Francisco, CA USA

[18] Univ Washington, Sch Med, Dept Pediat, Seattle Childrens Hosp, Seattle, WA 98195 USA

[19] Duke Univ, Med Ctr, Dept Pediat, Durham, NC 27710 USA

[20] Univ Paris Sud, Pediat & Adolescent Oncol, Gustave Roussy Inst, Villejuif, France

[21] Clin La Source, Inst Radiol, Lausanne, Switzerland

来源：

EUROPEAN JOURNAL OF NUCLEAR MEDICINE AND MOLECULAR IMAGING | 2018年 / 45卷 / 02期

关键词：

High-risk neuroblastoma; MIBG; SIOPEN score; STAGE; 4; NEUROBLASTOMA; STEM-CELL TRANSPLANTATION; HIGH-DOSE CHEMOTHERAPY; INDUCTION CHEMOTHERAPY; MULTIVARIATE-ANALYSIS; RANDOMIZED-TRIAL; CHILDREN; AGE; SCINTIGRAPHY; MELPHALAN;

D O I：

10.1007/s00259-017-3829-7

中图分类号：

R8 [特种医学]; R445 [影像诊断学];

学科分类号：

1002 ; 100207 ; 1009 ;

摘要：

Background Validation of the prognostic value of the SIOPEN mIBG skeletal scoring system in two independent stage 4, mIBG avid, high-risk neuroblastoma populations. Results The semi-quantitative SIOPEN score evaluates skeletal meta-iodobenzylguanidine (mIBG) uptake on a 0-6 scale in 12 anatomical regions. Evaluable mIBG scans from 216 COG-A3973 and 341 SIOPEN/HR-NBL1 trial patients were reviewed pre- and post-induction chemotherapy. The prognostic value of skeletal scores for 5-year event free survival (5 yr.-EFS) was tested in the source and validation cohorts. At diagnosis, both cohorts showed a gradual non-linear increase in risk with cumulative scores. Several approaches were explored to test the relationship between score and EFS. Ultimately, a cutoff score of <= 3 was the most useful predictor across trials. A SIOPEN score <= 3 pre-induction was found in 15% SIOPEN patients and in 22% of COG patients and increased post-induction to 60% in SIOPEN patients and to 73% in COG patients. Baseline 5 yr.-EFS rates in the SIOPEN/HR-NBL1 cohort for scores <= 3 were 47% +/- 7% versus 26% +/- 3% for higher scores at diagnosis (p < 0.007) and 36% +/- 4% versus 14% +/- 4% (p < 0.001) for scores obtained post-induction. The COG-A3973 showed 5 yr.-EFS rates for scores <= 3 of 51% +/- 7% versus 34% +/- 4% for higher scores (p < 0.001) at diagnosis and 43% +/- 5% versus 16% +/- 6% (p = 0.004) for post-induction scores. Hazard ratios (HR) significantly favoured patients with scores <= 3 after adjustment for age and MYCN-amplification. Optimal outcomes were recorded in patients who achieved complete skeletal response. Conclusions Validation in two independent cohorts confirms the prognostic value of the SIOPEN skeletal score. In particular, patients with an absolute SIOPEN score > 3 after induction have very poor outcomes and should be considered for alternative therapeutic strategies.

引用

页码：292 / 305

页数：14

共 32 条

[1]

[Anonymous], 2004, ANAL SURVIVAL DATA C

[2] REVISIONS OF THE INTERNATIONAL CRITERIA FOR NEUROBLASTOMA DIAGNOSIS, STAGING, AND RESPONSE TO TREATMENT [J].

BRODEUR, GM ;

PRITCHARD, J ;

BERTHOLD, F ;

CARLSEN, NLT ;

CASTEL, V ;

CASTLEBERRY, RP ;

DEBERNARDI, B ;

EVANS, AE ;

FAVROT, M ;

HEDBORG, F ;

KANEKO, M ;

KEMSHEAD, J ;

LAMPERT, F ;

LEE, REJ ;

LOOK, AT ;

PEARSON, ADJ ;

PHILIP, T ;

ROALD, B ;

SAWADA, T ;

SEEGER, RC ;

TSUCHIDA, Y ;

VOUTE, PA .

JOURNAL OF CLINICAL ONCOLOGY, 1993, 11 (08) :1466-1477

[3] Iodine-123 Metaiodobenzylguanidine Scintigraphy Scoring Allows Prediction of Outcome in Patients With Stage 4 Neuroblastoma: Results of the Cologne Interscore Comparison Study [J].

Decarolis, Boris ;

Schneider, Christina ;

Hero, Barbara ;

Simon, Thorsten ;

Volland, Ruth ;

Roels, Frederik ;

Dietlein, Markus ;

Berthold, Frank ;

Schmidt, Matthias .

JOURNAL OF CLINICAL ONCOLOGY, 2013, 31 (07) :944-951

[4] A phase II study of topotecan with vincristine and doxorubicin in children with recurrent/refractory neuroblastoma [J].

Garaventa, A ;

Luksch, R ;

Biasotti, S ;

Severi, G ;

Pizzitola, MR ;

Viscardi, E ;

Prete, A ;

Mastrangelo, S ;

Podda, M ;

Haupt, R ;

De Bernardi, B .

CANCER, 2003, 98 (11) :2488-2494

[5] Prognostic factors in metastatic neuroblastoma in patients over 1 year of age treated with high-dose chemotherapy and stem cell transplantation:: a multivariate analysis in 218 patients treated in a single institution [J].

Hartmann, O ;

Valteau-Couanet, D ;

Vassal, G ;

Lapierre, V ;

Brugières, L ;

Delgado, R ;

Couanet, D ;

Lumbroso, J ;

Benhamou, E .

BONE MARROW TRANSPLANTATION, 1999, 23 (08) :789-795

[6] Time-dependent ROC curves for censored survival data and a diagnostic marker [J].

Heagerty, PJ ;

Lumley, T ;

Pepe, MS .

BIOMETRICS, 2000, 56 (02) :337-344

[7] Survival model predictive accuracy and ROC curves [J].

Heagerty, PJ ;

Zheng, YY .

BIOMETRICS, 2005, 61 (01) :92-105

[8]

Heagerty PJ, 2013, SurvivalROC: Time-dependent ROC curve estimation from censored survival data

[9] Gaining more flexibility in Cox proportional hazards regression models with cubic spline functions [J].

Heinzl, H ;

Kaider, A .

COMPUTER METHODS AND PROGRAMS IN BIOMEDICINE, 1997, 54 (03) :201-208

[10] NONPARAMETRIC-ESTIMATION FROM INCOMPLETE OBSERVATIONS [J].

KAPLAN, EL ;

MEIER, P .

JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION, 1958, 53 (282) :457-481

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