Table 1.
Selection of larger data sets for patients with AT/RT in consistent registries and clinical trials
| Reference Time |
n | Age | M+ | Surgery (GTR) | Chemotherapy | Radiotherapy | Outcome | Comment |
|---|---|---|---|---|---|---|---|---|
| J. Hilden 200413 n.s. |
42 | 12 ≥ 3 y 20 < 3 y |
n = 9 | 20 | COG 99703 n = 8; CCG 9921 n = 6 and individual; n = 16 i.th. therapy; n = 13 HDCT |
n = 9 tumor bed; n = 4 CSI; various doses |
≥3 y: median EFS 16 mo; <2 y: median EFS 7.75 mo 2–3 y: median EFS 10.5 mo |
14 long-term survivors; GTR and older age prognostic |
| T. Tekautz 200512 1984–2003 |
31 | 9 ≥ 3 y 22 < 3 y |
6/31 | 21 | Non-uniform: ≥3 y, n = 7 SJMB96: <3 y, n = 7 BB98 and various others |
<3 y, n = 2 local, n = 1 CSI + boost ≥3 y, n = 7 CSI + boost |
<3 y: 2-y EFS ∼11% ± 6%; 2-y OS ∼17% ± 8% ≥3 y: 2-y EFS ∼78% ± 14%; 2-y OS ∼89% ± 11% |
Age >3 y prognostic; both long-term survivors received radiation |
| S. Chi 200933 2004–2006 |
20 | Median 26 mo 2.4 mo–9.5 y |
6/20 | 10 | IRS III–like | 11 conformal 4 CSI 4 none 1 off study 54 Gy focal, 36 Gy CSI + boost |
2-y PFS 53% ± 13% 2-y OS 70% ± 10% |
Only prospective phase II trial exclusively for AT/RT |
| K. von Hoff 201147 1988–2004 |
56 | Median 1.2 y 0.1–14 y | 26/56 | 18 | HIT medulloblastoma protocols |
n = 15 on primary therapy n = 14 at relapse 10/29 focal 19/29 CSI focal: 44.5–59.4 Gy CSI: 23.4–36.8 Gy |
3-y EFS: 13% ± 5% 3-y OS: 22% ± 6% |
Retrospective analysis of medulloblastoma cohort; age, achievement of CR, and M-disease prognostic |
| C. Dufour 2012116 1998–2008 |
58 | 38 ≤ 2 y | 17/58 10 < 2 y |
27 18 ≤ 2 y |
n = 24 ATRT04; n = 9 baby SFOP; n = 11 HDCT | Radiation in all but baby SFOP n = 16 7 ≤ 2 y |
Median OS 9 mo 1-y EFS: 17% 1-y OS: 41% |
Age <2 y, M+ disease and strong claudin; 6 staining negative prognosticators |
| L. Lafay-Cousin 201231 1995–2007 |
50 | 12 ≥ 3 y 21 = 1–3 y 17 < 1 y |
19/50 | 15 | 22 conventional; 18 HDCT eg, baby brain, IRS III–like, ICE; n = 9 anthracyclines |
21 as part of initial regimen; 6 at relapse |
2-y OS: 36.4 ± 7.7 median survival 9.6 mo in <1 y; 19.1 mo ≥ 3 y |
6/12 survivor no radiation HDCT: 2-y OS: 47.9% ± 12.1% convent.: 2-y OS 27.3% ± 9.5% |
| I. Slavc 201454 1992–2012 |
22 A: 9 B: 13 |
A: 24 mo B: 30 mo median age |
A: 4 B: 3 |
A: 5 B: 5 |
Cohort A: MUV (IRS III–like + HD-MTX + HDCT) cohort B: HIT-SKK, PEI, and others |
A: focal in all B: 3 focal, 4 CSI, 6 none |
Cohort A: 5-y OS 100%; 5-y EFS 88.9% ± 10.5% cohort B: 5-y OS and EFS: 28.8% ± 13.1% |
various i.th. regimens |
| K. Bartelheim submitted 2015 2005–2009 |
12 < 1 y; 11 @1–3 y; 8 ≥ 3 y | 6 | 10 | Rhabdoid 2007 = IRS III–like | <18 mo and in localized disease focal 54 Gy in CSI ≥ 18 mo 24 Gy + boost |
6-y OS: 46% ± 0.10% and 6-y EFS 45% ± 0.09% |
Germline mutation in 29%; age, achievement of CR prognostic |
Abbreviations: M+, metastatic; n.s., not specified; i.th., intrathecal; SFOP, French Society of Pediatric Oncology; HIT-SKK, Therapieprotokoll für Säuglinge und Kleinkinder mit Hirntumoren [Brain Tumor Radiotherapy for Infants and Toddlers with Medulloblastoma]; PEI, percutaneous ethanol injection; COG, Children's Oncology Group; SJMB, St Jude Medulloblastoma trial; ICE, ifosfamide/carboplatin/etoposide; IRS III, intergroup rhabdomypsarcoma study III.