Pediatric Cancer Clinical Research

The Center for Pediatric Hematology and Oncology is actively participating in clinical trials and translational research. A significant number of our clinical trials are through collaborative efforts with the Children's Oncology Group (COG), the Pediatric Brain Tumor Consortium (PBTC), the New England Retinoblastoma Group and the Hodgkin Lymphoma Consortium.


Research Summary

COG is an association of more than 200 childhood cancer treatment and research centers located primarily throughout North America that cooperates in clinical and translational research programs related to all types of cancer of infants, children, and young adults. The COG trials include Phase I, II and III studies. The PBTC was formed by the NCI in 1999 to improve the treatment of primary brain tumors in children. The 10 academic centers and childrens hospitals that are members of the PBTC conduct novel Phase 1 and Phase 2 clinical evaluations of new therapeutic drugs and biologics, and treatment delivery strategies. The PBTC and COG also conduct research on tumor specimens obtained from children on clinical trials. We also develop and coordinate innovative imaging studies through the above groups, as well as with ACRIN.

Members of our group have leadership roles in COG, PBTC and the Retinoblastoma and Hodgkin consortia. Recently activated new protocols have focused on novel treatments for children with brain tumors, soft tissue and bone sarcomas, retinoblastoma, acute leukemias and Hodgkin lymphomas. A recently completed COG led by Dr. David Ebb evaluated the toxicity and efficacy of the combination of Herceptin® and standard chemotherapy for children with previously untreated metastatic osteosarcoma.

Our Pediatric Hematology and Oncology Program is committed to developing more effective and less toxic treatments for children with malignancies. In collaboration with our colleagues in Pediatric Radiation Oncology at Massachusetts General Hospital Cancer Center and the Massachusetts Eye & Ear Infirmary, we have developed novel clinical trials combining proton beam radiation with chemotherapy for children with medulloblastoma, retinoblastoma and bone and soft tissue sarcomas. One of the major objectives of these trials is to reduce both the acute and long-term sequelae of therapy by using protons instead of standard radiation techniques (photons or electrons). Compared to other forms of radiation therapy, protons do not have an exit path and, therefore, spare toxicity to many normal tissues in a growing child. A large multidisciplinary team is involved in these studies, including subspecialists in ophthalmology, pediatric neurosurgery, pediatric oncology, pediatric radiation oncology, pediatric neurology, pediatric endocrinology, speech and language, and neuropsychology.

During the past 12 years, we have been active members of a Hodgkin lymphoma consortium involving investigators at Stanford, St. Jude, Dana-Farber and Maine Children's Cancer Program. We have completed several studies that were successful in reducing therapy and complications and maintaining excellent survival for children with favorable stages of Hodgkin's disease. The current Hodgkin's clinical trials are designed to further reduce therapy by omitting involved field irradiation for children with favorable stage disease who achieve a complete response to upfront chemotherapy and consolidating chemotherapy to 12 weeks in combination with low-dose involved field irradiation for children with advanced stage disease.

We have studies focusing on quality of life for our young cancer patients. An active trial is testing the feasibility of early discharge from the hospital for children who are admitted with fever and neutropenia. This protocol assesses the medical outcomes, financial implications, and, very importantly, how families cope with this extra burden of home care. Other studies are evaluating the neurocognitive sequelae of brain irradiation and correlating the clinical data with novel imaging modalities. These studies are done in collaboration with pediatric neurology, radiology and developmental psychology.

In keeping with the theme of improving quality of life, we have developed a school outreach program for our patients at all levels of education. A team of nurses and social workers visits the schools of our patients and presents relevant information to the faculty and classmates. An ongoing, productive dialogue ensues often with follow-up visits and bi-directional recommendations. This program has been enormously successful in maintaining a sense of normalcy for our patients.

Group Members


1 Hudson MM, Krasin M, Link MP, Donaldson SS, Billups C, Merchant TE, Kun L, Billet AL, Kaste S, Tarbell NJ, Howard S, Friedmann AM, Hurwitz CA, Young JA, Marcus KC, Rai S, Cowan T, Weinstein HJ Risk-adapted, combined-modality therapy with VAMP/COP and response-based, involved-field radiation for unfavorable pediatric Hodgkin's disease. J Clin Oncol. 11/15/2004; 22(22); 4541-50.

2 Laver JH, Kraveka JM, Hutchison RE, Chang M, Kepner J, Schwenn M, Tarbell N, Desai S, Weitzman S, Weinstein HJ, Murphy SB Advanced-stage large-cell lymphoma in children and adolescents: results of a randomized trial incorporating intermediate-dose methotrexate and high-dose cytarabine in the maintenance phase of the APO regimen: a Pediatric Oncology Group phase III trial. J Clin Oncol. 01/20/2005; 23(3); 541-7.

3 Ravindranath Y, Chang M, Steuber CP, Becton D, Dahl G, Civin C, Camitta B, Carroll A, Raimondi SC, Weinstein HJ, Pediatric Oncology Group (POG) studies of acute myeloid leukemia (AML): a review of four consecutive childhood AML trials conducted between 1981 and 2000. Leukemia. 11/23/2005; 19(12); 2101-16.

4 Massey GV, Zipursky A, Chang MN, Doyle JJ, Nasim S, Taub JW, Ravindranath Y, Dahl G, Weinstein HJ, A prospective study of the natural history of transient leukemia (TL) in neonates with Down syndrome (DS): Children's Oncology Group (COG) study POG-9481. Blood. 06/06/2006; 107(12); 4606-13.

5 Donaldson SS, Link MP, Weinstein HJ, Rai SN, Brain S, Billett AL, Hurwitz CA, Krasin M, Kun LE, Marcus KC, Tarbell NJ, Young JA, Hudson MM Final results of a prospective clinical trial with VAMP and low-dose involved-field radiation for children with low-risk Hodgkin's disease. J Clin Oncol. 01/19/2007; 25(3); 332-7.

6 Friedmann AM, Weinstein HJ The evolving standard of care for hodgkin lymphoma. J Pediatr Hematol Oncol. 03/31/2008; 30(2); 121-3.

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