Clinical and molecular characterization of isolated M1 disease in pediatric medulloblastoma: experience from the German HIT-MED studies
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Clinical and molecular characterization of isolated M1 disease in pediatric medulloblastoma: experience from the German HIT-MED studies. / Obrecht, Denise; Mynarek, Martin; Hagel, Christian; Kwiecien, Robert; Spohn, Michael; Bockmayr, Michael; Bison, Brigitte; Pfister, Stefan M; Jones, David T W; Sturm, Dominik; von Deimling, Andreas; Sahm, Felix; von Hoff, Katja; Juhnke, B-Ole; Benesch, Martin; Gerber, Nicolas U; Friedrich, Carsten; von Bueren, André O; Kortmann, Rolf-Dieter; Schwarz, Rudolf; Pietsch, Torsten; Fleischhack, Gudrun; Schüller, Ulrich; Rutkowski, Stefan.
in: J NEURO-ONCOL, Jahrgang 157, Nr. 1, 03.2022, S. 37-48.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Clinical and molecular characterization of isolated M1 disease in pediatric medulloblastoma: experience from the German HIT-MED studies
AU - Obrecht, Denise
AU - Mynarek, Martin
AU - Hagel, Christian
AU - Kwiecien, Robert
AU - Spohn, Michael
AU - Bockmayr, Michael
AU - Bison, Brigitte
AU - Pfister, Stefan M
AU - Jones, David T W
AU - Sturm, Dominik
AU - von Deimling, Andreas
AU - Sahm, Felix
AU - von Hoff, Katja
AU - Juhnke, B-Ole
AU - Benesch, Martin
AU - Gerber, Nicolas U
AU - Friedrich, Carsten
AU - von Bueren, André O
AU - Kortmann, Rolf-Dieter
AU - Schwarz, Rudolf
AU - Pietsch, Torsten
AU - Fleischhack, Gudrun
AU - Schüller, Ulrich
AU - Rutkowski, Stefan
N1 - © 2022. The Author(s).
PY - 2022/3
Y1 - 2022/3
N2 - PURPOSE: To evaluate the clinical impact of isolated spread of medulloblastoma cells into cerebrospinal fluid without additional macroscopic metastases (M1-only).METHODS: The HIT-MED database was searched for pediatric patients with M1-only medulloblastoma diagnosed from 2000 to 2019. Corresponding clinical and molecular data was evaluated. Treatment was stratified by age and changed over time for older patients.RESULTS: 70 patients with centrally reviewed M1-only disease were identified. Clinical data was available for all and molecular data for 45/70 cases. 91% were non-WNT/non-SHH medulloblastoma (Grp3/4). 5-year PFS for 52 patients ≥ 4 years was 59.4 (± 7.1) %, receiving either upfront craniospinal irradiation (CSI) or SKK-sandwich chemotherapy (CT). Outcomes did not differ between these strategies (5-year PFS: CSI 61.7 ± 9.9%, SKK-CT 56.7 ± 6.1%). For patients < 4 years (n = 18), 5-year PFS was 50.0 (± 13.2) %. M1-persistence occurred exclusively using postoperative CT and was a strong negative predictive factor (pPFS/OS < 0.01). Patients with additional clinical or molecular high-risk (HR) characteristics had worse outcomes (5-year PFS 42.7 ± 10.6% vs. 64.0 ± 7.0%, p = 0.03). In n = 22 patients ≥ 4 years with full molecular information and without additional HR characteristics, risk classification by molecular subtyping had an effect on 5-year PFS (HR 16.7 ± 15.2%, SR 77.8 ± 13.9%; p = 0.01).CONCLUSIONS: Our results confirm that M1-only is a high-risk condition, and further underline the importance of CSF staging. Specific risk stratification of affected patients needs attention in future discussions for trials and treatment recommendations. Future patients without contraindications may benefit from upfront CSI by sparing risks related to higher cumulative CT applied in sandwich regimen.
AB - PURPOSE: To evaluate the clinical impact of isolated spread of medulloblastoma cells into cerebrospinal fluid without additional macroscopic metastases (M1-only).METHODS: The HIT-MED database was searched for pediatric patients with M1-only medulloblastoma diagnosed from 2000 to 2019. Corresponding clinical and molecular data was evaluated. Treatment was stratified by age and changed over time for older patients.RESULTS: 70 patients with centrally reviewed M1-only disease were identified. Clinical data was available for all and molecular data for 45/70 cases. 91% were non-WNT/non-SHH medulloblastoma (Grp3/4). 5-year PFS for 52 patients ≥ 4 years was 59.4 (± 7.1) %, receiving either upfront craniospinal irradiation (CSI) or SKK-sandwich chemotherapy (CT). Outcomes did not differ between these strategies (5-year PFS: CSI 61.7 ± 9.9%, SKK-CT 56.7 ± 6.1%). For patients < 4 years (n = 18), 5-year PFS was 50.0 (± 13.2) %. M1-persistence occurred exclusively using postoperative CT and was a strong negative predictive factor (pPFS/OS < 0.01). Patients with additional clinical or molecular high-risk (HR) characteristics had worse outcomes (5-year PFS 42.7 ± 10.6% vs. 64.0 ± 7.0%, p = 0.03). In n = 22 patients ≥ 4 years with full molecular information and without additional HR characteristics, risk classification by molecular subtyping had an effect on 5-year PFS (HR 16.7 ± 15.2%, SR 77.8 ± 13.9%; p = 0.01).CONCLUSIONS: Our results confirm that M1-only is a high-risk condition, and further underline the importance of CSF staging. Specific risk stratification of affected patients needs attention in future discussions for trials and treatment recommendations. Future patients without contraindications may benefit from upfront CSI by sparing risks related to higher cumulative CT applied in sandwich regimen.
U2 - 10.1007/s11060-021-03913-5
DO - 10.1007/s11060-021-03913-5
M3 - SCORING: Journal article
C2 - 35190934
VL - 157
SP - 37
EP - 48
JO - J NEURO-ONCOL
JF - J NEURO-ONCOL
SN - 0167-594X
IS - 1
ER -
