Surgical Management of Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic

Christopher B. Weldon, Arin L. Madenci, Sosipatros A. Boikos, Katherine A. Janeway, Suzanne George, Margaret Von Mehren, Alberto S. Pappo, Joshua D. Schiffman, Jennifer Wright, Jonathan Trent, Karel Pacak, Constantine A. Stratakis, Lee J. Helman, Michael P La Quaglia

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Abstract

Purpose Wild-type gastrointestinal stromal tumors (WT-GISTs) that lack KIT or PDGFRA mutations represent a unique subtype of GIST that predominantly affects children. We sought to determine the effect on event-free survival (EFS) of staging variables, extent of resection, and repeat resection of tumors. Methods In 2008, a WT-GIST clinic was established at the National Cancer Institute, allowing the development of a large clinical database. We included participants who underwent resection of WT-GIST. Associations with EFS (ie, freedom from disease progression or recurrence) were evaluated using the Kaplan-Meier method and Cox proportional hazards modeling. Results Among 76 participants with WT-GISTs, the median follow-up was 4.1 years. Overall EFS (6 SE) was 72.6 6 5.4% at 1 year, 57.6 6 6.2% at 2 years, 23.7 6 6.0% at 5 years, and 16.3 6 5.5% at 10 years postoperatively. Hazard of disease progression or recurrence was significantly increased for patients with metastatic disease (adjusted hazard ratio [AHR], 2.3; 95% CI, 1.0 to 5.1; P 5 .04) and . 5 mitoses per 50 high-power fields (AHR, 2.5; 95% CI, 1.1 to 6.0; P 5 .03), whereas there was no significant effect of negative microscopic resection margins (AHR, 0.9; 95% CI, 0.4 to 2.2; P 5 0.86). There was no association between type of gastric resection (ie, anatomic v partial/wedge) and EFS (P 5 .67). Repeated resection after the initial resection was significantly associated with decreasing postoperative EFS (P , .01). Five patients (6%) died after initial enrollment in 2008. Conclusion WT-GIST is an indolent disease, and most patients survive with disease progression. We found no improvement in EFS with more extensive or serial resections. Disease progression or recurrence may be more closely related to tumor biology than surgical management. These data suggest that resections for WT-GISTs be restricted to the initial procedure and that subsequent resections be performed only to address symptoms such as obstruction or bleeding.

Original languageEnglish (US)
Pages (from-to)523-528
Number of pages6
JournalJournal of Clinical Oncology
Volume35
Issue number5
DOIs
StatePublished - Feb 10 2017

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Gastrointestinal Stromal Tumors
National Institutes of Health (U.S.)
Disease-Free Survival
Pediatrics
Disease Progression
Recurrence
National Cancer Institute (U.S.)
Mitosis
Neoplasms
Stomach
Databases
Hemorrhage
Mutation

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Surgical Management of Wild-Type Gastrointestinal Stromal Tumors : A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic. / Weldon, Christopher B.; Madenci, Arin L.; Boikos, Sosipatros A.; Janeway, Katherine A.; George, Suzanne; Von Mehren, Margaret; Pappo, Alberto S.; Schiffman, Joshua D.; Wright, Jennifer; Trent, Jonathan; Pacak, Karel; Stratakis, Constantine A.; Helman, Lee J.; Quaglia, Michael P La.

In: Journal of Clinical Oncology, Vol. 35, No. 5, 10.02.2017, p. 523-528.

Research output: Contribution to journalArticle

Weldon, CB, Madenci, AL, Boikos, SA, Janeway, KA, George, S, Von Mehren, M, Pappo, AS, Schiffman, JD, Wright, J, Trent, J, Pacak, K, Stratakis, CA, Helman, LJ & Quaglia, MPL 2017, 'Surgical Management of Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic', Journal of Clinical Oncology, vol. 35, no. 5, pp. 523-528. https://doi.org/10.1200/JCO.2016.68.6733
Weldon, Christopher B. ; Madenci, Arin L. ; Boikos, Sosipatros A. ; Janeway, Katherine A. ; George, Suzanne ; Von Mehren, Margaret ; Pappo, Alberto S. ; Schiffman, Joshua D. ; Wright, Jennifer ; Trent, Jonathan ; Pacak, Karel ; Stratakis, Constantine A. ; Helman, Lee J. ; Quaglia, Michael P La. / Surgical Management of Wild-Type Gastrointestinal Stromal Tumors : A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic. In: Journal of Clinical Oncology. 2017 ; Vol. 35, No. 5. pp. 523-528.
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title = "Surgical Management of Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic",
abstract = "Purpose Wild-type gastrointestinal stromal tumors (WT-GISTs) that lack KIT or PDGFRA mutations represent a unique subtype of GIST that predominantly affects children. We sought to determine the effect on event-free survival (EFS) of staging variables, extent of resection, and repeat resection of tumors. Methods In 2008, a WT-GIST clinic was established at the National Cancer Institute, allowing the development of a large clinical database. We included participants who underwent resection of WT-GIST. Associations with EFS (ie, freedom from disease progression or recurrence) were evaluated using the Kaplan-Meier method and Cox proportional hazards modeling. Results Among 76 participants with WT-GISTs, the median follow-up was 4.1 years. Overall EFS (6 SE) was 72.6 6 5.4{\%} at 1 year, 57.6 6 6.2{\%} at 2 years, 23.7 6 6.0{\%} at 5 years, and 16.3 6 5.5{\%} at 10 years postoperatively. Hazard of disease progression or recurrence was significantly increased for patients with metastatic disease (adjusted hazard ratio [AHR], 2.3; 95{\%} CI, 1.0 to 5.1; P 5 .04) and . 5 mitoses per 50 high-power fields (AHR, 2.5; 95{\%} CI, 1.1 to 6.0; P 5 .03), whereas there was no significant effect of negative microscopic resection margins (AHR, 0.9; 95{\%} CI, 0.4 to 2.2; P 5 0.86). There was no association between type of gastric resection (ie, anatomic v partial/wedge) and EFS (P 5 .67). Repeated resection after the initial resection was significantly associated with decreasing postoperative EFS (P , .01). Five patients (6{\%}) died after initial enrollment in 2008. Conclusion WT-GIST is an indolent disease, and most patients survive with disease progression. We found no improvement in EFS with more extensive or serial resections. Disease progression or recurrence may be more closely related to tumor biology than surgical management. These data suggest that resections for WT-GISTs be restricted to the initial procedure and that subsequent resections be performed only to address symptoms such as obstruction or bleeding.",
author = "Weldon, {Christopher B.} and Madenci, {Arin L.} and Boikos, {Sosipatros A.} and Janeway, {Katherine A.} and Suzanne George and {Von Mehren}, Margaret and Pappo, {Alberto S.} and Schiffman, {Joshua D.} and Jennifer Wright and Jonathan Trent and Karel Pacak and Stratakis, {Constantine A.} and Helman, {Lee J.} and Quaglia, {Michael P La}",
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language = "English (US)",
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T1 - Surgical Management of Wild-Type Gastrointestinal Stromal Tumors

T2 - A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic

AU - Weldon, Christopher B.

AU - Madenci, Arin L.

AU - Boikos, Sosipatros A.

AU - Janeway, Katherine A.

AU - George, Suzanne

AU - Von Mehren, Margaret

AU - Pappo, Alberto S.

AU - Schiffman, Joshua D.

AU - Wright, Jennifer

AU - Trent, Jonathan

AU - Pacak, Karel

AU - Stratakis, Constantine A.

AU - Helman, Lee J.

AU - Quaglia, Michael P La

PY - 2017/2/10

Y1 - 2017/2/10

N2 - Purpose Wild-type gastrointestinal stromal tumors (WT-GISTs) that lack KIT or PDGFRA mutations represent a unique subtype of GIST that predominantly affects children. We sought to determine the effect on event-free survival (EFS) of staging variables, extent of resection, and repeat resection of tumors. Methods In 2008, a WT-GIST clinic was established at the National Cancer Institute, allowing the development of a large clinical database. We included participants who underwent resection of WT-GIST. Associations with EFS (ie, freedom from disease progression or recurrence) were evaluated using the Kaplan-Meier method and Cox proportional hazards modeling. Results Among 76 participants with WT-GISTs, the median follow-up was 4.1 years. Overall EFS (6 SE) was 72.6 6 5.4% at 1 year, 57.6 6 6.2% at 2 years, 23.7 6 6.0% at 5 years, and 16.3 6 5.5% at 10 years postoperatively. Hazard of disease progression or recurrence was significantly increased for patients with metastatic disease (adjusted hazard ratio [AHR], 2.3; 95% CI, 1.0 to 5.1; P 5 .04) and . 5 mitoses per 50 high-power fields (AHR, 2.5; 95% CI, 1.1 to 6.0; P 5 .03), whereas there was no significant effect of negative microscopic resection margins (AHR, 0.9; 95% CI, 0.4 to 2.2; P 5 0.86). There was no association between type of gastric resection (ie, anatomic v partial/wedge) and EFS (P 5 .67). Repeated resection after the initial resection was significantly associated with decreasing postoperative EFS (P , .01). Five patients (6%) died after initial enrollment in 2008. Conclusion WT-GIST is an indolent disease, and most patients survive with disease progression. We found no improvement in EFS with more extensive or serial resections. Disease progression or recurrence may be more closely related to tumor biology than surgical management. These data suggest that resections for WT-GISTs be restricted to the initial procedure and that subsequent resections be performed only to address symptoms such as obstruction or bleeding.

AB - Purpose Wild-type gastrointestinal stromal tumors (WT-GISTs) that lack KIT or PDGFRA mutations represent a unique subtype of GIST that predominantly affects children. We sought to determine the effect on event-free survival (EFS) of staging variables, extent of resection, and repeat resection of tumors. Methods In 2008, a WT-GIST clinic was established at the National Cancer Institute, allowing the development of a large clinical database. We included participants who underwent resection of WT-GIST. Associations with EFS (ie, freedom from disease progression or recurrence) were evaluated using the Kaplan-Meier method and Cox proportional hazards modeling. Results Among 76 participants with WT-GISTs, the median follow-up was 4.1 years. Overall EFS (6 SE) was 72.6 6 5.4% at 1 year, 57.6 6 6.2% at 2 years, 23.7 6 6.0% at 5 years, and 16.3 6 5.5% at 10 years postoperatively. Hazard of disease progression or recurrence was significantly increased for patients with metastatic disease (adjusted hazard ratio [AHR], 2.3; 95% CI, 1.0 to 5.1; P 5 .04) and . 5 mitoses per 50 high-power fields (AHR, 2.5; 95% CI, 1.1 to 6.0; P 5 .03), whereas there was no significant effect of negative microscopic resection margins (AHR, 0.9; 95% CI, 0.4 to 2.2; P 5 0.86). There was no association between type of gastric resection (ie, anatomic v partial/wedge) and EFS (P 5 .67). Repeated resection after the initial resection was significantly associated with decreasing postoperative EFS (P , .01). Five patients (6%) died after initial enrollment in 2008. Conclusion WT-GIST is an indolent disease, and most patients survive with disease progression. We found no improvement in EFS with more extensive or serial resections. Disease progression or recurrence may be more closely related to tumor biology than surgical management. These data suggest that resections for WT-GISTs be restricted to the initial procedure and that subsequent resections be performed only to address symptoms such as obstruction or bleeding.

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