The addition of autologous stem cell transplantation to neoadjuvant chemotherapy, radiation, and HIPEC for patients with unresectable desmoplastic small round cell tumor: a single center case series.

Amir Siddiqui,Grace Z Mak,Michele L Nassin,John M Cunningham,James L Labelle,Mark A Applebaum,Navin Pinto

doi:10.1097/ij9.0000000000000095

Abstract

Desmoplastic small round cell tumor (DSRCT) is a rare, highly aggressive malignancy primarily affecting children and young adults. Although modest improvements have been gained by intensification of chemotherapy and radiation, survival of patients with DSRCT remains poor, particularly in those with unresectable or disseminated disease. We report 3 pediatric patients who were treated with a combination of therapy including chemotherapy, surgical debulking, hyperthermic intraperitoneal chemotherapy, whole abdominal irradiation, and autologous hematopoietic stem cell transplantation following busulfan and melphalan conditioning. We find that this approach is well tolerated and may offer improved survival in patients with DSRCT.

Highlights

Desmoplastic small round cell tumor (DSRCT) is a rare, highly aggressive mesenchymal tumor with dismal patient outcomes
The use of these specific chemotherapy agents is considered off label as they are not approved for DSRCT, they are commonly used in this patient population
Biopsy revealed small round blue cells and cytogenetics confirmed the EWSR1-WT1 translocation. He was diagnosed with stage 4 DSRCT and received 6 cycles of vincristine, etoposide, ifosfamide, and doxorubicin (VIDE) followed by 1 cycle of vincristine, dactinomycin and ifosfamide (VAI) during surgical planning

Summary

Materials and methods

An IRB approved (IRB 12362A) retrospective review was performed to identify patients diagnosed with DSRCT and treated consecutively at the University of Chicago Comer Children’s Hospital, an urban academic tertiary care center, between 2013 and 2018. International Journal of Surgery Oncology preoperative goal of complete resection when possible. This occurred in the operating room (OR), under general anesthesia in the supine position. All patients considered eligible for this therapy were required to be chemotherapy responsive and have good performance status upon entering the procedure so no additional preintervention therapy was required to optimize the patients before the OR. The use of these specific chemotherapy agents is considered off label as they are not approved for DSRCT, they are commonly used in this patient population. Data is reported in line with PROCESS 2018 criteria[15]

Results

Discussion

Ethical approval