Treatment

It is important to find doctors experienced in GIST. This experience comes in two basic forms: experts in pediatric cancers and experts in adult GIST cancer. Both have unique perspectives and experiences. The best approach might be when doctors from both fields collaborate with each other. See Where to go for Treatment.

Surgical removal of the tumor(s) has historically been the primary treatment for Pediatric GIST 1,2,3,4. The types of operations have historically varied from a simple local excision to a total gastrectomy2. In some cases, surgery provides long-term relief. In other cases, tumors return, either locally (near the original site, such as another stomach tumor(s)), or distant from the original site (metastases).

Recently, dramatic success has occurred in the treatment of adults with metastatic or inoperable GISTs (in cases where surgery is not possible). The genetic defects that cause adult GIST (KIT and PDGFRA mutations) can now be targeted with a drug called Gleevec® (also known as imatinib and Glivec®). While this drug (and a second generation inhibitor, Sutent®) is very effective in adult GIST, the vast majority of Pediatric GISTs do not have the same types of mutations. Response data is limited, but the early ancedotal indications are that Gleevec is not as effective in pediatric GIST as it is in adult GIST. Some preliminary data (3 patients reported at the 2006 American Society of Clinical Oncologists (ASCO) meeting) show some activity for Sutent 5. In March, 2009, updated information was published on this group and 3 additional pediatric GIST patients (6 patients total)6.

1. Gastrointestinal Stromal Tumors in Children and Young Adults.
A Clinicopathologic, Molecular, and Genomic Study of 15 Cases and Review of the Literature. J Pediatr Hematol Oncol Volume 27, Number 4, April 2005. Sonam Prakash, MD, Lisa Sarran, MS, Nicholas Socci, PhD, Ronald P. DeMatteo, MD, Jonathan Eisenstat, MD, Alba M. Greco, MD, Robert G. Maki, MD, PhD,{ Leonard H. Wexler, MD,k Michael P. LaQuaglia, MD, Peter Besmer, PhD, and Cristina R. Antonescu, MD

2. Gastrointestinal Stromal Tumors of the Stomach in Children and Young Adults
A Clinicopathologic, Immunohistochemical, and Molecular Genetic Study of 44 Cases With Long-Term Follow-Up and Review of the Literature.
Am J Surg Pathol Volume 29, Number 10, October 2005 Markku Miettinen, MD,* Jerzy Lasota, MD,* and Leslie H. Sobin, MD†

3. Clinical and Molecular Characteristics of Pediatric Gastrointestinal Stromal Turmos (GISTs)
Victoria E. Price, Maria Zielenska, Susan Chilton-MacNeill, Charles R. Smith and Alberto S. Pappo Pediatr Blood Cancer

4. Pediatric Gastrointestinal Stromal Tumors and Leiomyosarcoma
The St. Jude Children's Research Hospital Experience and a Review of the Lieterature CANCER July 1, 2004 / Volume 101 / Number 1

5. Sunitinib treatment of pediatric metastatic GIST after failure of imatinib
K. A. Janeway, D. C. Matthews, J. E. Butrynski, G. Z. D'Amato, S. Agresta, C. Garrett, C. L. Corless, K. H. Albritton, G. D. Demetri Journal of Clinical Oncology, 2006 ASCO Annual Meeting Proceedings Part I. Vol 24, No. 18S (June 20 Supplement), 2006: 9519

6. Sunitinib treatment in pediatric patients with advanced GIST following failure of imatinib
Katherne A. Janeway, MD, Karen H. Albritton, MD, Annick D. Van Den Abbeele, MD, Gina Z. D'Amato, MD, Paolo Pedrazzoli, MD, Salvatore Siena, MD, Joel Picus, MD, James E. Butrynski, MD, Marcus Schlemmer, MD, Michael C. Heinrich, MD, George D. Demetri, MD, Pdeatr Blood Cancer