RARE-31. RECURRENT CHOROID PLEXUS CARCINOMA IN THE SETTING OF LI-FRAUMENI SYNDROME: REPORT OF TWO CHILDREN MANAGED WITH INTENSIVE RE-INDUCTION AND MARROW-ABLATIVE CONSOLIDATION CHEMOTHERAPY WITHOUT IRRADIATION FOLLOWED BY MOLECULARLY-TARGETED BIOLOGICAL THERAPY
| Autor: | Christopher R. Pierson, Daniel R. Boue, Jeffrey R. Leonard, Elaine R. Mardis, Jonathan L. Finlay, Claire Heinerich, Elizabeth Varga, Margaret Shatara, Diana S Osorio, Mohamed S. AbdelBaki, Catherine E. Cottrell, Maciej Ciołkowski, Paweł Kowalczyk, Richard K. Wilson, Rolla Abu-Arja, Iwona Filipek, Jeremy Jones |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
Oncology
Cancer Research medicine.medical_specialty Cyclophosphamide business.industry Consolidation Chemotherapy Choroid plexus carcinoma medicine.disease Chemotherapy regimen Transplantation Li–Fraumeni syndrome Internal medicine Carcinoma AcademicSubjects/MED00300 Medicine AcademicSubjects/MED00310 Choroid plexus Neurology (clinical) business Craniopharyngioma and Rare Tumors medicine.drug |
| Zdroj: | Neuro-Oncology |
| ISSN: | 1523-5866 1522-8517 |
| DOI: | 10.1093/neuonc/noaa222.741 |
| Popis: | BACKGROUND The optimal management for children with recurrent choroid plexus carcinoma (CPC), is not established. We report two children with germline TP53 mutations, whose CPC relapses were managed with marrow-ablative chemotherapy and oral biologically-targeted therapies. PATIENTS: Patient A: A 17 months old male presented with non-metastatic bilateral CPC. A de novo mosaic germline TP53 mutation was identified. After near-total resections, 16 months of standard chemotherapy were administered; 18 months later, localized tumor growth developed, again near-totally resected. Two cycles of re-induction chemotherapy were administered followed by three cycles of thiotepa/carboplatin with autologous hematopoietic cell rescue (AuHCR) and subsequently 21 months of sirolimus and thalidomide, continuing without residual or recurrent disease. Patient B: A 30 months old male presented with left lateral ventricular non-metastatic CPC. A de novo TP53 germline mutation was identified. Following sub-total resection, craniospinal irradiation with boost was administered followed by eight cycles of standard chemotherapy; 18 months later, localized recurrence developed; gross total resection was followed by 15 months of standard dose chemotherapies; four months thereafter, a second local recurrence developed, again gross totally resected. He then received one cycle of high-dose cyclophosphamide followed by three cycles of thiotepa/carboplatin with AuHCR. Subsequently he received sirolimus and thalidomide for 12 months, complicated by progressive pancytopenia. A small localized CPC recurrence was noted, gross totally resected, concomitant with myelodysplastic syndrome; he underwent an allogeneic matched unrelated donor marrow transplantation. CONCLUSIONS Marrow-ablative chemotherapy with post-transplant targeted biological therapy may afford durable survival for select children with recurrent CPC. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|