Tandem high-dose chemotherapy with topotecan–thiotepa–carboplatin and melphalan–etoposide–carboplatin regimens for pediatric high-risk brain tumors

Choi, Jung Yoon; Kang, Hyoung Jin; Hong, Kyung Taek; Hong, Che Ry; Lee, Yun Jeong; Park, June Dong; Kim, Seung Ki; Wang, Kyu Chang; Kim, Il Han; Park, Shin Young; Choi, Young Hun; Cheon, Jung Eun; Park, Kyung Duk; Shin, Hee Young

doi:10.1007/s10147-019-01517-8

Cited by 10 publications

(7 citation statements)

References 37 publications

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“…Additionally, in a retrospective study with limited small sample size with embryonal tumors, HDMTX combined with dose-intensified multiagent chemotherapy was used; it was suggested that in children with high-risk brain tumors who have a poor outcome, High-dose chemotherapy (HDC) and autologous stem-cell transplantation (auto-SCT) could be an option in treatment and promising to improve survival rates. 33 A retrospective study from China indicated that aggressive surgery as first-line therapy in PB and younger age was associated with poorer prognosis. 34 In our series, the patients who lived longer were older than the others.…”

Section: Discussionmentioning

confidence: 99%

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Görgün¹,

Koç²,

Kebudi³

et al. 2021

Turk J Pediatr

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Pineoblastoma (PB) is a rare embryonal tumor of the pineal gland. 1 Histologically, PB are classified as WHO grade IV tumors. 2,3 While they typically appear radiographically as focal enhancing mass, PB can also be locally invasive and spread outside the pineal region through the subarachnoid space. 4 In this study, we assessed the demographics, treatment, late effects and outcome of PB patients who were diagnosed and treated in the Istanbul University Oncology Institute (IUOI) during 1990-2012. Material and MethodsDuring 1990-2012, 516 children with brain tumors were diagnosed and treated in the IUOI. Among these patients six (1.16 %) were diagnosed with PB. Demographics, age of diagnosis, first complaint, tumor region, diagnosis type, seeding metastasis to the spinal axis or cerebrospinal fluid (CSF), treatment and survival of these patients were evaluated retrospectively from patient records. All patients had cranial and spinal axis MRI with contrast at diagnosis and during follow-up.

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Section: Discussionmentioning

confidence: 99%

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Görgün¹,

Koç²,

Kebudi³

et al. 2021

Turk J Pediatr

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“…We used a melphalan/etoposide/carboplatin regimen, which was different from the studies that conducted HDC in OSA. This regimen has already been used for a variety of childhood cancers and is familiar to pediatricians ( 20 – 22 ). All of these medications are known to be effective in OSA, and we have confirmed through a previously reported pilot study that the melphalan/etoposide/carboplatin regimen showed good treatment results, especially in localized OSA patients with low-degree necrosis ( 14 ).…”

Section: Discussionmentioning

confidence: 99%

Favorable outcome of high-dose chemotherapy and autologous hematopoietic stem cell transplantation in patients with nonmetastatic osteosarcoma and low-degree necrosis

et al. 2022

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BackgroundA low-degree tumor necrosis after neoadjuvant chemotherapy is a poor prognostic factor for osteosarcoma (OSA). However, the role of high-dose chemotherapy (HDC) and autologous hematopoietic stem cell transplantation in OSA remains controversial. We analyzed the treatment outcomes and prognostic factors of nonmetastatic OSA and compared the HDC and conventional chemotherapy (CC) outcomes of patients with <90% necrosis after neoadjuvant chemotherapy.MethodsWe retrospectively evaluated patients with OSA treated at the Seoul National University Children’s Hospital from 2000 to 2020. Totally, 113 patients with non-metastatic OSA at diagnosis were included. The majority were treated with cisplatin, doxorubicin, and methotrexate as neoadjuvant chemotherapy. This was continued when the postoperative necrosis rate was >90% (good response [GR]), whereas most cases with <90% (poor response [PR]) were changed to chemotherapy. The HDC regimen was composed of melphalan, etoposide, and carboplatin.ResultsThe median age at diagnosis was 12.6 years (range, 5.0–20.3), and 61.9% of patients were men. The 5-year event-free survival (EFS) and overall survival (OS) rates were 75.8% and 91.5%, respectively. Among these, 59 and 44 patients were included in the GR and PR groups, respectively. The GR group had a better 5-year EFS rate than the PR group (82.4% vs. 67.3%, p=0.071). Age at diagnosis, sex, tumor site, type of neoadjuvant chemotherapy, and degree of tumor necrosis were not different between the PR-HDC (n=24) and PR-CC (n=20) groups. The 5-year EFS and OS rates in the PR-HDC (n=24) and PR-CC (n=20) groups were 78.6% and 53.6% (p=0.065) and 100% and 76.9% (p=0.024), respectively. In the Cox regression analysis, the PR-CC group (hazard ratio, 4.95; p=0.004) and age ≥12 years (hazard ratio, 2.68; p=0.024) were significant risk factors for 5-year EFS.ConclusionsHDC showed favorable outcomes in patients with non-metastatic OSA and <90% necrosis after neoadjuvant chemotherapy.

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“…The efficacy of MEC HDCT for malignant rhabdoid tumors of the kidney was not proven, but this entity is associated with high therapy resistance and a very poor prognosis [26]. The role of MEC in brain tumors is limited and the protocol is usually included as a part of the tandem HDCT strategy [27]. MEC HDCT in osteosarcoma did not show any clear advantage, and the megatherapy strategy is considered ineffective in this indication [28].…”

Section: Discussionmentioning

confidence: 99%

Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis

Ussowicz

Mielcarek‐Siedziuk

Musiał³

et al. 2020

Cancers

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Pediatric germ cell tumors (GCTs) are a group of chemosensitive malignancies with a 90% curability rate. We report a series of children with relapsing or therapy-resistant GCT treated with melphalan–etoposide–carboplatin high-dose chemotherapy (HDCT) and autologous stem cell transplantation. This consisted of 18 children, either with GCTs after relapse (nine patients) or with an unsatisfactory response to first-line chemotherapy (nine patients), who underwent HDCT. The HDCT regimens MEC1 (carboplatin 1500 mg/m2, etoposide 1800 mg/m2, and melphalan 140 mg/m2) and MEC2 (carboplatin 800 mg/m2, etoposide 800 mg/m2, and melphalan 140 mg/m2) were each used in nine patients. The median observation time was 81 months, the 5-year overall survival (OS) was 76%, and the event-free survival (EFS) was 70.8%. Non-relapse mortality was 0%, and four patients died after HDCT due to progression of the malignancy. No difference in OS or EFS was noted between the MEC1 and MEC2 protocols. The 5-year OS and 5-year EFS were higher in children treated with autologous stem cell transplantation before the age of four years. The presence of metastatic disease or time of HDCT consolidation during first/subsequent line chemotherapy did not affect patient survival. The melphalan–etoposide–carboplatin protocol is feasible in pediatric GCT, but is associated with potentially life-threatening complications. In conclusion, the use of HDCT must be examined in well-designed clinical trials, and the identification of patients who can benefit from this approach is critical to avoid overtreatment.

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Tandem high-dose chemotherapy with topotecan–thiotepa–carboplatin and melphalan–etoposide–carboplatin regimens for pediatric high-risk brain tumors

Cited by 10 publications

References 37 publications

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Clinical characteristics, late effects and outcomes in pineoblastomas in children: a single center experience

Favorable outcome of high-dose chemotherapy and autologous hematopoietic stem cell transplantation in patients with nonmetastatic osteosarcoma and low-degree necrosis

Melphalan, Etoposide, and Carboplatin Megatherapy with Autologous Stem Cell Transplantation in Children with Relapsing or Therapy-Resistant Extracranial Germ-Cell Tumors—A Retrospective Analysis

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