Pediatric Cancers

Rituxan for Opsoclonus Myoclonus Syndrome – pro

Rituxan has been reported to alleviate the myoclonus opsoclonus syndrome in case reports and series. Presumably there is an immunologic mechanism underlying this observation. Approximately 50 % of patients with OMS have an associated neuroblastoma or associated infections have also been reported.  There is no specific diagnostic biomarker for OMS. Other treatments include as ACTH, corticosteroids, cyclophosphamide and/or intravenous immunoglobulin,

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Pediatric testicular cancer – pro

Malignant germ cell tumors (MGCT) account for 3% to 4% of childhood malignancies (< 15 years of age). Before the advent of multimodal therapy, children with MGCT could expect poor outcomes. Subsequent therapy was based on the larger adult experience with epithelial ovarian cancer because there was a paucity of clinical trials in pediatric patients. Cyclophosphamide-based therapy improved the outcome for patients with localized MGC but for patients

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Topotecan and cytoxan for neuroblastoma – pro

Topotecan and cyclophosphamide are both active drugs in neuroblastoma and various combinations of these two drugs with or without other additional drugs have been used in a number of phase II trials. There are no FDA approved drugs for neuroblastoma and therefore this regimen and the individual drugs are considered off-label and experimental but it is medically necessary  based on phase II studies, one phase III study  and the lack of alternatives.

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3f8 and leukine for neuroblastoma – pro

The proposed therapy is a clinical phase II trial performed at MSKCC. Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining colony-stimulating factors, such as sargramostim, with monoclonal antibodies may be an effective treatment for advanced neuroblastoma. It is a Phase II trial to study the effectiveness of monoclonal antibody 3F8 plus sargramostim

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Ch14.18 for neuroblastoma – pro

Ch14.18 is a chimeric human/murine anti-GD2 antibody, lyses neuroblastoma cells. This study determined the maximum tolerable dose (MTD) and toxicity of ch14.18 given in combination with interleukin-2 (IL-2) after high-dose chemotherapy (HDC)/stem-cell rescue (SCR). Biologic correlates including ch14.18 levels, soluble IL-2 receptor levels, and human antichimeric antibody (HACA) activity were evaluated. A Phase I study enrolled 25 patients. The MTD

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Etoposide, doxorubicin, carboplatin for intermediate risk neuroblastoma – pro

With current treatments, patients with low and intermediate risk disease have an excellent prognosis with cure rates above 90%. There are no FDA approved drugs for neuroblastoma and therefore  this regimen and the individual drugs are considered off-label  but it is medically necessary  based on phase II studies. The most common combination of drugs to treat neuroblastoma consists of carboplatin (or cisplatin), cyclophosphamide, doxorubicin, and

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CAMT – pro

Congenital amegakaryocytic thrombocytopenia (CAMT) is clinically characterized by thrombocytopenia presenting at birth in a child without congenital or skeletal malformations, reduced or absent bone marrow megakaryocytes, and eventual progression to bone marrow failure. The problem is an absence of a receptor for thrombopoietin. There is not curative option at thsi time other than allogeneic transpalnt. This is a rare condition for which there are

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Allogeneic Stem Cell Transplantation in Childhood ALL – pro

ALL is the most common cancer diagnosed in children and represents almost 25% of cancers in children younger than 15 years. Complete remission of disease is now typically achieved with pediatric chemotherapy regimens in approximately 95% of children with ALL, with up to 85% long-term survival rates. Those who relapse have few good options; retreatment rarely produces a cure. Three randomized controlled trials (RCTs) that compared outcomes of hematopoietic

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Neuroblastoma – Stem Cell Transplantation – pro

Lay Summary: Neuroblastoma is the only PNET in which the value of stem cell transpalntation in certain situations has been proven. Primitive neuroepithelial tumors include medulloblastoma, neuroblastoma arising in the central nervous system, ependymoblastoma, or pineoblastoma. All show a similar histology and are principally distinguished by their site of origin. Essentially, medulloblastoma may be considered a cerebellar or posterior fossa PNET

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