Desmoplastic small round cell tumor: from state of the art to future clinical prospects

Figures & data

Table 1. Selected studies on desmoplastic small round cell tumor.

Author	Series	Treatment	Outcome
Kushner et al, 1996, MSKCC, New York, USA [9]	Prospective, single-institution study; Study period: not reported; Number of cases: 12; Median age 14 years (range 7–22)	multiagent intensive chemotherapy (P6 protocol), 11/12 had surgery, 4/12 had local radiotherapy, 4/12 had ASCT	5 pts alive with NED (10, 13, 14, 34, 39 months after diagnosis)
Goodman et al, 2002, MSKCC, New York, USA [10]	Retrospective, single-institution study; Study period: 1992–2001; Number of cases: 21; Median age, years: 16.5 (range 8–34)	multiagent chemotherapy (alkylator-based), surgery, WAP-RT ±ASCT	median PFS 19 mos, 3-year PFS 19%; median OS 32 months, 3-year OS 48%
Lal et al, 2005, MSKCC, New York, USA [11]	Retrospective, single-institution study; Study period: 1972–2003; Number of cases: 66; Median age, years: 19 (range 7–58)	44% had trimodal treatment including chemotherapy (P6 protocol), surgery and WAP-RT	3-year OS 44%, 5-year OS 15%; 3-year OS 55% for pts given trimodal therapy vs 27% for pts with one modality missing; multimodal therapy correlated with survival, while distant metastases did not
Hayes-Jordan et al, 2010, MDACC, Houston, USA [12]	Retrospective, single-institution study; Study period: 1995–2008; Number of cases: 24; Median age, years: 12	8/24 (33%) had surgery + HIPEC	3-year OS 71% for pts who were treated with HIPEC vs 26% for patients who were not
Bisogno et al, 2010, Italian Pediatric Oncology Association study (AIEOP-RMS4.99) [13]	Prospective, multi-institution study; Study period: 1999–2008; Number of cases: 14; Median age, years: 9 (range 2–18)	all pts had multiagent chemotherapy (10 IVADo, 4 CEVAIE), followed by ASCT in 10/14, 12/14 had surgery, 6/14 had WAP-RT	3-year EFS 15.5%; 3-year OS 38.9%
Cook et al, 2012, (CIBMTR) [14]	Retrospective study on registry data; Study period: 1999–2007; Number of cases: 36; Median age, years: 19 (8–46)	35/36 had ASCT (i.e. thiotepa or etoposide or melphalan or cyclophosphamide)	median OS 31 mos, 1-year OS 83%, 3-year OS 40%; 3-year OS was 57% for pts in CR before ASCT (36% of the cases) vs 28% for others
Philippe-Chomette P et al. 2012, French national series [7]	Retrospective national multicenter study; Study period: 1995–2006; Number of cases: 38; Median age, year: 13.2 (4–29.7)	all pts had multiagent chemotherapy, 9 had limited surgical resection, 22 had extensive resection, 14 had ASCT	3-year EFS 14.4%; 3-year OS 50.5%; no prognostic factors (radiotherapy, ASCT, extend of surgery)
Pinnix et al, 2012, MDACC, Houston, USA [15]	Retrospective, single-institution study; Study period: 2006–2010; Number of cases: 8; Median age, years: 11.5 (range 5–20)	multiagent chemotherapy (VAC/IE), surgery, WAP-RT (IMRT)	median PFS 8.7 months; 1/8 alive with NED after 20 months
Desai et al, 2013, MSKCC, New York, USA [16]	Retrospective, single-institution study; Study period: 1992–2011; Number of cases: 31; Median age, years: 19 (range 7–32)	all pts had multiagent chemotherapy, surgery and WAP-RT (22 2D-RT, 9 IMRT)	3-year PFS 24%; 3-year OS 50%; OS correlated with distant metastases
Wong et al, 2013, Royal Marsden Hospital, London, and Addenbrooke’s Hospital, Cambridge, UK [17]	Retrospective, multi- institution study; Study period: 1991–2012; Number of cases: 41; Median age, years: 27 (range 16–45)	multiagent chemotherapy (i.e. VIDE, IVADo, VAC/IE), 20% had surgery, 15% had WAP-RT	median PFS 4 months; median OS 16 mos, 3-year OS 27%, 5-year OS 16%; VIDE chemotherapy conferred longest TTP; surgery for localized disease and radiotherapy for metastatic disease correlated with improved OS
Hayes-Jordan et al, 2013, MDACC, Houston, USA [18]	Retrospective, single-institution study; Study period: 2006–2011; Number of cases: 26; Median age, years: 19 (range 6–53)	all pts had multiagent chemotherapy, surgery, HIPEC, ± local radiotherapy	median OS 31 months for pts who had complete surgery, 12.8 months for pts who had partial resection; 1-year DFS 42% in cases of abdominal disease vs 0% in cases of extra-abdominal disease; no benefit of HIPEC in pts with extra-abdominal disease; HIPEC morbidity lower in children
Zhang et al, 2015, Shandong Cancer Hospital, Jinan, China [19]	Retrospective, single-institution study; Study period: 2004–2014; Number of cases: 11; Median age, years: 31.4 (range 14–64)	multiagent chemotherapy (IAP, CAP), 5/11 had local radiotherapy	median PFS 8.8 months, 3-year PFS 27%; median OS 29 months, 3-year OS 36.4%, 5-year OS 10%; median OS 40.8 months for pts given radiotherapy vs 19.2 months for the others
Honoré et al, 2015, IGR, Villejuif, France [4]	Retrospective, single-institution study; Study period: 1991–2013; Number of cases: 38; Median age, years: 27 (range 13–57)	multiagent chemotherapy (alkylating/anthracyclines-based, i.e. AI, VAC), 61% had surgery, 21% had WAP-RT	median DFS 15.5 months, median OS 37.7 months; 2/38 (5%) alive with NED after 32 and 37 months; absence of extra-peritoneal disease, complete surgery, and radiotherapy were prognostic factors; no benefit of surgery for extra-peritoneal metastatic disease
Desai et al, 2015, MSKCC, New York, USA [20]	Prospective single-institution study; Study period: 1993–2004; Number of cases: 19; Median age, years: 18.5 (range 10–42)	multiagent chemotherapy followed by myeloablative chemotherapy (carboplatin-thiotepa ± topotecan), with autologous stem cell transplant (in patients with chemoresponsive DSRCT); 17/19 had radiotherapy	5-year EFS 11% 5-year OS 16%
Atallah et al, 2016, French sarcoma group [21]	Retrospective, multi-institution study; Study period: 1991–2014; Number of cases: 107; Median age, years: 25 (range 4–58)	36% of pts had chemotherapy, surgery and WAP-RT, 34% had chemotherapy and surgery	median OS 40.3 months and 3-year OS 63.1% for pts who had surgery and radiotherapy, and 28.3 months and 48.5%, respectively, for pts who had surgery without radiotherapy
Osborne et al, 2016, MDACC, Houston, USA [22]	Retrospective, single institution study; Study period: 2006–2014; Number of cases: 32; Median age, years: 18 (range 5–50)	multiagent chemotherapy, surgery HIPEC, WAP-RT (23/32 IMRT)	median DFS 10 months, 3-year DFS 9.9%; median OS 60 months, 3-years OS 64%
Honoré et al, 2017, French Network databases [18]	Retrospective, multi-institution, nation-wide study; Study period: 1991–2015; Number of cases: 107 (pts with extra-abdominal metastases were excluded); Median age, years: 22 (range 3–57)	48% had multimodal therapy with chemotherapy, surgery and WAP-RT, 23% had HIPEC	median DFS 21 months, 2-year DFS 30%, 5-year DFS 12%; median OS 42 months, 2-year OS 72%, 5-year OS 19%; whole-abdomen radiotherapy correlated with DFS
Stiles et al, 2018, Tennessee National Cancer Data Base, USA [23]	Retrospective study on registry data; Study period: 2004–2014; Number of cases: 125; Median age, years: 21	82% had multiagent chemotherapy, 60% had surgery, 17% had radiotherapy, 6% had ASCT	median OS 28 months, 3-year OS 29%, 5-year OS 10%; multimodal treatment correlated with survival; residual postoperative macroscopic disease increased risk of mortality
Subbiah et al, 2018, MDACC, Houston, USA [24]	Retrospective, single-institution study; Study period: 1990–2016; Number of cases: 187; Median age, years: 23 (range 0–53)	98% had multiagent chemotherapy (i.e. VAC/IE, VIDE, P6 protocol), 57% had surgery, 44% had HIPEC, 6% had ASCT, 49% had trimodal therapy with chemotherapy, surgery and WAP-RT	median OS 35 months, 3-year OS 48%; whole-abdomen radiotherapy did not improve OS
Hayes-Jordan et al, 2018, MDACC, Houston, USA [25]	Prospective, single-institution, phase 2 trial; Study period: 2012–2013; Number of cases: 14 (pts with extra-abdominal metastases were excluded); Median age, years: 21	14/14 had multiagent chemotherapy + complete surgery + HIPEC + WAP-RT	median RFS 15 months; median OS 58.4 months, 3-year OS 79%
Scheer et al, 2019, German Pediatric Group CWS [6]	Retrospective cooperative study; Study period: 1997–2015; Number of cases: 60; Median age, years: 15 (6–38)	all pts had multiagent chemotherapy (i.e. P6 protocol, VAIA, CEVAIE), 35% had surgery, 10% had HIPEC, 33% had radiotherapy, 15% had ASCT	3-year EFS 11%, 3-year OS 30%; VAIA chemotherapy correlated with longer EFS
Honoré et al, 2019, French Sarcoma Group [26]	Retrospective, multi-institution, nation-wide study; Study period: 1991–2018; Number of cases: 100; Median age, years: 25 (range 3–59)	80% had up-front multiagent chemotherapy (i.e. P6 protocol, VAC/IE, VIDE), 71% had surgery, followed by HIPEC in 28% of them, and by WAP-RT in 50%	median PFS 11 months, 3-year PFS 7%, 5-year PFS 6%; median OS 25 months, 3-year OS 35%, 5-year OS 4%; 5/100 alive with NED; prognostic factors: complete surgery, female sex, whole-abdomen radiotherapy
Campos et al, 2020, AC Camargo Cancer Center, São Paulo, Brazil [27]	Retrospective, single-institution study; Study period: 2007–2020; Number of cases: 19; Median age, years: 26 (15–41)	all pts had multiagent chemotherapy (i.e. VAC/IE, VAC, AI), 58% had surgery, 26% had HIPEC, 21% had WAP-RT	median PFS 8.7 months; median OS 27 months, 3-year OS 38%, 5-year 12%
Xiang et al, 2020, Chinese databases [8]	Retrospective study on registry data; Study period: 2000–2015; Number of cases: 104; Median age, years: 24 (range 15–54)	88% had chemotherapy, 66% had surgery, 23% had radiotherapy	median OS 26 months, 3-year OS 33%; prognostic factors: surgical patterns, metastatic status, and adjuvant chemotherapy
Liu et al, 2021, Dana-Farber Cancer Institute and Boston Children’s Hospital [28]	Retrospective, single-institution study; Study period: 2014–2019; Number of cases: 6; Median age, years: 15 (range 3–16)	multiagent dose-density chemotherapy (VIT/VDC/IE), followed by surgery and WAP-RT	3/6 pts alive with NED 21, 46 and 60 months after diagnosis; 2-year OS 75%
Ferrari et al, 2021, INT, Milano, Italy [29]	Retrospective, single-institution study; Study period: 2017–2018; Number of cases: 3; Median age, years: 16 (range 10–20)	multiagent dose-density chemotherapy (IrIVA/IVAd/IVE), surgery, WAP-RT, maintenance chemotherapy (vinorelbine and low-dose oral cyclophosphamide)	dose-density treatment with irinotecan was feasible; 1/3 alive with NED 37 months after diagnosis, 2 alive with disease after abdominal relapse
Giani, et al. 2023, INT, Milan [30]	Retrospective, single-institution study; Study period: 2000–2021; Number of cases: 38; Median age, years: 25 (range 7–64)	multiagent chemotherapy ± surgery (71%) ± HIPEC (26%) ± WAP-RT (24%) ± high-dose chemotherapy (13%) ± maintenance chemotherapy (32%)	median-EFS 15 months; median-OS 37 months; long-term survivors had no liver/extra-abdominal disease, and were treated with complete surgery, and possibly WAP-RT and maintenance therapy

Note: Legend: pts = patients; OS = overall survival; PFS = progression-free survival; PPFS = peritoneal progression-free survival; DFS = disease-free survival; RFS = relapse-free survival; EFS = event-free survival; TTP = time to progression; NED = no evidence of disease; mos = months; WAP-RT = whole abdominopelvic radiotherapy; HIPEC = hyperthermic intraperitoneal chemotherapy; 2D = two-dimensional; IMRT = intensity-modulated radiation therapy; ASCT = autologous stem cell transplantation; P6 Protocol = HD-CAV (cyclophosphamide, doxorubicin, vincristine) alternating with ifosfamide, etoposide; VIDE = vincristine, ifosfamide, doxorubicin, etoposide; IVADo = ifosfamide, vincristine, actinomycin D, doxorubicin; VAC/IE = vincristine, adriamycin, cyclophosphamide/ifosfamide, etoposide; IAP = ifosfamide, doxorubicin, cisplatin; CAP = cyclophosphamide, doxorubicin, cisplatin; AI = doxorubicin, ifosfamide; VIT/VDC/IE = vincristine, irinotecan, temozolomide/vincristine, doxorubicin, cyclophosphamide/ifosfamide, etoposide; IrIVA = irinotecan, ifosfamide, vincristine, actinomycin-D; IVAd = ifosfamide, vincristine, adriamycin; IVE = ifosfamide, vincristine, etoposide; CEVAIE = ifosfamide, vincristine, actinomycin D, carboplatin, epirubicin, etoposide; EAM = extra-abdominal metastases, CR = complete response; MSKCC = Memorial Sloan Kettering Cancer Center; MDACC = Anderson Cancer Center; IGR = Institut Gustave Roussy; CWS = Cooperative Weichteilsarkom Studiengruppe (German Cooperative Group); INT = Istituto Nazionale dei Tumori.

Table 2. Potential therapeutic agents for desmoplastic small round cell tumor.

Agent	Author	Findings
Trabectidin	Lopez-Gonzalez et al, 2011 [73]	1 patient with PR, 8 months of PFS
	Brunetti et al, 2014 [74]	1 patient with SD, 4 months of PFS
	Frezza et al, 2014 [75]	2 patients with SD, 4 months of PFS
	Verret et al, 2017 [76]	6 patients, 2 with SD and 4 with PD, 1–4 months of PFS
Irinotecan	Ambar et al. [66]	irinotecan + vincristine (plus radiotherapy) 1 patient with PR, 26 months of PFS
Irinotecan and trabectidin	Ferrari et al, 2022 [77]	trabectidin + irinotecan: 2 patients, 1 with CR, 1 with SD
Temsirolimus	Thijs et al, 2010 [78]	1 patient with SD, 9 months of PFS
	Naing et al, 2012 [79]	temsirolimus + cixutumumab: 3 patients, 2 with PR, 5 months of PFS
	Tarek et al, 2018 [80]	temsirolimus + vinorelbine + cyclophosphamide: 5 patients with PR, 8.6 months of PFS
Vinorelbine	Ferrari et al, 2007 [81]	vinorelbine + cyclophosphamide: 2 patients with PR, 4 months of PFS
	Tarek et al, 2018 [71]	vinorelbine + cyclophosphamide + temsirolimus: 5 patients with PR, 8.6 months of PFS
Antiangiogenic agents	Bétrian et al, 2017 [82]	sunitinib, bevacizumab, sorafenib: 9 patients, 2 with PD, 7 with SD, 3 months of PFS
Pazopanib	Frezza et al, 2014 [75]	9 patients, 5 with SD, 2 with PD, 2 with PR, <16 months of PFS
	Menegaz et al, 2018 [83]	29 patients, 16 with SD, 1 with PD, 11 with PR, 5 months of PFS
Imatinib	De Sanctis et al, 2017 [84]	8 patients, 1 with SD, 7 with PD, 3 months of PFS
Ganitumab	Tap et al, 2012 [85]	16 patients, 6 with PR, 10 with SD, 19 months of PFS
Sunitinib	Italiano et al. 2013 [86]	8 patients, 3 with SD, 3 with PD, 2 with PR, 1–20 months of PFS
Eribulin	Emambux et al. 2017 [87]	3 patients, 2 with SD, 1 with PD, 2–9 months of PFS
Anlotinib	Chen et al, 2019 [88]	1 patient with PR
	Cheng et al, 2022 [89]	1 patient with PR
Apatinib	Tian et al, 2020 [82]	1 patient with PR
Androgen receptor pathway	Fine, 2007 [90]	bicalutamide + leuprorelin: 6 patients, 3 with SD, 3–4 months of PFS bicalutamide: 1 patient with PD, 2.5 months of PFS
Prexasertib	Slotkin, et al 2022 [91]	Prexasertib+irinotecan 19 patients, 9 with SD, 6 with PR, 3 with SD, 1 with PD

Note: Legend: PFS = progression-free survival; PD = progressive disease; SD = stable disease; PR = partial response; CR = complete remission.

Table 3. Trials recruiting patients with desmoplastic small round cell tumor as of October 2022.

Phase	Title/design/age criteria	Primary outcome	ClinicalTrials.gov Identifier
Phase I/II	Ramucirumab IV + Cyclophosphamide p.o. + Vinorelbine IV (experimental arm), versus Cyclophosphamide p.o. + Vinorelbine IV. Age inclusion criteria: 12 months −29 years	PFS	NCT04145349
Phase II	131 I-Omburtamab in Combination with External Beam Radiotherapy. Age inclusion criteria: >1 year	PFS	NCT04022213
Phase I/II	PBI-200 in Subjects with NTRK-Fusion-Positive Advanced or Metastatic Solid Tumors. Age inclusion criteria: >18 years	Safety and tolerability ORR	NCT04901806
Phase II	Trabectedin and Low-dose Radiation Therapy in Advanced/Metastatic Sarcomas. Age inclusion criteria: 16–75 years	ORR in irradiated nodules	NCT05131386
Phase I	LSD1 Inhibitor Seclidemstat (SP 2577) With and Without Topotecan and Cyclophosphamide in Patients with Relapsed or Refractory Ewing Sarcoma and Selected Sarcomas. Age inclusion criteria: 12 years	Safety and tolerability	NCT03600649
Phase I	B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients with Solid Tumors (3CAR), Age inclusion criteria: < 21 years	Safety of B7-H3-CAR T cells	NCT04897321
Phase I	EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults, Age inclusion criteria: 1–30 years	Safety and tolerability	NCT03618381
Phase I	B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults Age inclusion criteria: 0–26 years	Safety and tolerability	NCT04483778
Phase II	Multimodal Immune Characterization of RAre Soft Tissue Sarcoma – MIRAS Project from SARRA (SARcome RAre) Project of the French Sarcoma Group.Age inclusion criteria: >18 years	MFS for localized disease, PFS for metastatic disease	NCT03967834
Phase I	A Prospective Study of Heated Intra-Peritoneal Chemotherapy (HIPEC) with Doxorubicin and Cisplatin in Pediatric Patients with Pelvic and Abdominal Tumors. Age inclusion criteria: 1–25 years	Adverse events of surgery with HIPEC	NCT04213794
Phase II	A Study of the Drug I131-Omburtamab in People With Desmoplastic Small Round Cell Tumors and Other Solid Tumors in the Peritoneum	PFS	NCT04022213
Phase I/II	A Study of Repotrectinib in Combination With Chemotherapy in Children and Young Adults With Solid Tumor Cancer	Safety and tolerability	NCT05004116

Note: Legend:.

IV, intravenous; p.o., per os; PFS, progression-free survival; ORR, overall response rate; MFS, metastasis-free survival; HIPEC, Hyperthermic intraperitoneal chemotherapy; CAR T cell, chimeric antigen receptor T-cell.

Kushner BH, LaQuaglia MP, Wollner N, et al. Desmoplastic small round-cell tumor: prolonged progression-free survival with aggressive multimodality therapy. J Clin Oncol. 1996 May;14(5):1526–1531.

 PubMed Web of Science ®Google Scholar

Goodman KA, Wolden SL, la Quaglia MP, et al. Whole abdominopelvic radiotherapy for desmoplastic small round-cell tumor. Int J Radiat Oncol Biol Phys. 2002 Sep;54(1):170–176.

 PubMedGoogle Scholar

Lal DR, Su WT, Wolden SL, et al. Results of multimodal treatment for desmoplastic small round cell tumors. J Pediatr Surg. 2005 Jan;40(1):251–255.

 PubMed Web of Science ®Google Scholar

Hayes-Jordan A, Green H, Fitzgerald N, et al. Novel treatment for desmoplastic small round cell tumor: hyperthermic intraperitoneal perfusion. J Pediatr Surg. 2010 May;45(5):1000–1006.

 PubMedGoogle Scholar

Bisogno G, Ferrari A, Rosolen A, et al. Sequential intensified chemotherapy with stem cell rescue for children and adolescents with desmoplastic small round-cell tumor. Bone Marrow Transplant. 2010 May;45(5):907–911.

 PubMed Web of Science ®Google Scholar

Cook RJ, Wang Z, Arora M, et al. Clinical outcomes of patients with desmoplastic small round cell tumor of the peritoneum undergoing autologous HCT: a CIBMTR retrospective analysis. Bone Marrow Transplant. 2012 Nov 2;47(11):1455–1458.

 PubMedGoogle Scholar

Philippe-Chomette P, Kabbara N, Andre N, et al. Desmoplastic small round cell tumors with EWS-WT1 fusion transcript in children and young adults. Pediatr Blood Cancer. 2012 Jun;58(6):891–897.

 PubMedGoogle Scholar

Pinnix CC, Fontanilla HP, Hayes-Jordan A, et al. Whole abdominopelvic intensity-modulated radiation therapy for desmoplastic small round cell tumor after surgery. Int J Radiat Oncol Biol Phys. 2012 May 1;83(1):317–326.

 PubMedGoogle Scholar

Desai NB, Stein NF, LaQuaglia MP, et al. Reduced toxicity with intensity modulated radiation therapy (IMRT) for desmoplastic small round cell tumor (DSRCT): an update on the whole abdominopelvic radiation therapy (WAP-RT) experience. Int J Radiat Oncol Biol Phys. 2013 Jan 1;85(1):e67–72.

 PubMed Web of Science ®Google Scholar

Wong HH, Hatcher HM, Benson C, et al. Desmoplastic small round cell tumour: characteristics and prognostic factors of 41 patients and review of the literature. Clin Sarcoma Res. 2013 Dec 26;3(1):14.

 PubMedGoogle Scholar

Honoré C, Atallah V, Mir O, et al. Abdominal desmoplastic small round cell tumor without extraperitoneal metastases: is there a benefit for HIPEC after macroscopically complete cytoreductive surgery? PLoS ONE. 2017 Feb 24;12(2):e0171639.

 PubMedGoogle Scholar

Zhang S, Zhang Y, Yu YH, et al. Results of multimodal treatment for desmoplastic small round cell tumor of the abdomen and pelvis. Int J Clin Exp Med. 2015;8(6):9658–9666.

 PubMedGoogle Scholar

Honoré C, Amroun K, Vilcot L, et al. Abdominal desmoplastic small round cell tumor: multimodal treatment combining chemotherapy, surgery, and radiotherapy is the best option. Ann Surg Oncol. 2015 Apr 10;22(4):1073–1079.

 PubMed Web of Science ®Google Scholar

Forlenza CJ, Kushner BH, Kernan N, et al. Myeloablative chemotherapy with autologous stem cell transplant for desmoplastic small round cell tumor. Sarcoma 2015;2015:269197 [Epub 2015 Apr 7].

 PubMedGoogle Scholar

Atallah V, Honore C, Orbach D, et al. Role of adjuvant radiation therapy after surgery for abdominal desmoplastic small round cell tumors. Int J Radiat Oncol Biol Phys. 2016 Jul;95(4):1244–1253.

 PubMedGoogle Scholar

Osborne EM, Briere TM, Hayes-Jordan A, et al. Survival and toxicity following sequential multimodality treatment including whole abdominopelvic radiotherapy for patients with desmoplastic small round cell tumor. Radiother Oncol. 2016 Apr;119(1):40–44.

 PubMed Web of Science ®Google Scholar

Stiles ZE, Dickson PV, Glazer ES, et al. Desmoplastic small round cell tumor: a nationwide study of a rare sarcoma. J Surg Oncol. 2018 Jun;117(8):1759–1767.

 PubMedGoogle Scholar

Subbiah V, Lamhamedi-Cherradi SE, Cuglievan B, et al. Multimodality treatment of desmoplastic small round cell tumor: chemotherapy and complete cytoreductive surgery improve patient survival. Clin Cancer Res. 2018 Oct 1;24(19):4865–4873.

 PubMed Web of Science ®Google Scholar

Hayes-Jordan AA, Coakley BA, Green HL, et al. Desmoplastic small round cell tumor treated with cytoreductive surgery and hyperthermic intraperitoneal chemotherapy: results of a phase 2 trial. Ann Surg Oncol. 2018 Apr 30;25(4):872–877.

 PubMedGoogle Scholar

Scheer M, Vokuhl C, Blank B, et al. Desmoplastic small round cell tumors: multimodality treatment and new risk factors. Cancer Med. 2019 Feb;8(2):527–542.

 PubMed Web of Science ®Google Scholar

Honoré C, Delhorme JB, Nassif E, et al. Can we cure patients with abdominal desmoplastic small round cell tumor? Results of a retrospective multicentric study on 100 patients. Surg Oncol. 2019 Jun;29:107–112.

 PubMed Web of Science ®Google Scholar

Campos F, Coutinho DL, Silva MLG, et al. Clinical characteristics, management, and outcomes of 19 nonpediatric patients with desmoplastic small round cell tumor: a cohort of Brazilian patients. Sarcoma. 2020 Oct 28;2020:1–7.

 Google Scholar

Xiang T, Zhang SY, Wang SS, et al. A nationwide analysis of desmoplastic small round cell tumor. Medicine. 2020 Jul 24;99(30):e21337.

 PubMed Web of Science ®Google Scholar

Liu KX, Collins NB, Greenzang KA, et al. The use of interval‐compressed chemotherapy with the addition of vincristine, irinotecan, and temozolomide for pediatric patients with newly diagnosed desmoplastic small round cell tumor. Pediatr Blood Cancer. 2020 Oct 19;67(10). DOI:10.1002/pbc.28559

 Google Scholar

Bisogno G, Ferrari A, Tagarelli A, et al. Integrating irinotecan in standard chemotherapy: a novel dose‐density combination for high‐risk pediatric sarcomas. Pediatr Blood Cancer. 2021 Jul 10;68(7). DOI:10.1002/pbc.28951

 Google Scholar

Giani C, Radaelli S, Miceli R, et al. Long-term survivors with desmoplastic small round cell tumor (DSRCT): results from a retrospective single-institution case series analysis. Cancer Med. 2023 Mar 23. DOI:10.1002/cam4.5829

 PubMedGoogle Scholar

López-González A, Cantos B, Tejerina E, et al. Activity of trabectidin in desmoplastic small round cell tumor. Med Oncol. 2011 Dec 7;28(S1):644–646.

 Google Scholar

Brunetti AE, Delcuratolo S, Lorusso V, et al. Third-line trabectedin for a metastatic desmoplastic small round cell tumour treated with multimodal therapy. Anticancer Res. 2014;34(7):3683–3688.

 PubMed Web of Science ®Google Scholar

Frezza AM, Whelan JS, Dileo P. Trabectedin for desmoplastic small round cell tumours: a possible treatment option? Clin Sarcoma Res. 2014 Dec 25;4(1):3.

 PubMedGoogle Scholar

Verret B, Honore C, Dumont S, et al. Trabectedin in advanced desmoplastic round cell tumors. Anticancer Drugs. 2017 Jan;28(1):116–119.

 PubMed Web of Science ®Google Scholar

Ambar NBD, de Seixas Alves MT, Lederman HM, et al. Irinotecan and vincristine for the treatment of refractory desmoplastic small round cell tumor in a developing country: a case report. J Med Case Rep. 2019 Dec 10;13(1):77.

 PubMedGoogle Scholar

Ferrari A, Chiaravalli S, Bergamaschi L, et al. Trabectedin-irinotecan, a potentially promising combination in relapsed desmoplastic small round cell tumor: report of two cases. J Chemother. 2023 Apr;35(2):163–167.

 PubMed Web of Science ®Google Scholar

Thijs AMJ, van der Graaf WTA, van Herpen CML. Temsirolimus for metastatic desmoplastic small round cell tumor. Pediatr Blood Cancer. 2010 Dec 15;55(7):1431–1432.

 PubMed Web of Science ®Google Scholar

Naing A, LoRusso P, Fu S, et al. Insulin growth factor-receptor (IGF-1R) antibody cixutumumab combined with the mTOR inhibitor temsirolimus in patients with refractory ewing’s sarcoma family tumors. Clin Cancer Res. 2012 May 1;18(9):2625–2631.

 PubMed Web of Science ®Google Scholar

Tarek N, Hayes-Jordan A, Salvador L, et al. Recurrent desmoplastic small round cell tumor responding to an mTOR inhibitor containing regimen. Pediatr Blood Cancer. 2018 Jan;65(1):e26768. DOI:10.1002/pbc.26768

 Google Scholar

Ferrari A, Grosso F, Stacchiotti S, et al. Response to vinorelbine and low-dose cyclophosphamide chemotherapy in two patients with desmoplastic small round cell tumor. Pediatr Blood Cancer. 2007 Nov;49(6):864–866.

 PubMedGoogle Scholar

Bertuzzi A, Castagna L, Nozza A, et al. High-dose chemotherapy in poor-prognosis adult small round-cell tumors: clinical and molecular results from a prospective study. J Clin Oncol. 2002 Apr 15;20(8):2181–2188.

 PubMedGoogle Scholar

Tian Y, Cheng X, Li Y. Chemotherapy combined with apatinib for the treatment of desmoplastic small round cell tumors: a case report. J Cancer Res Ther. 2020;16(5):1177.

 PubMedGoogle Scholar

Menegaz BA, Cuglievan B, Benson J, et al. Clinical activity of pazopanib in patients with advanced desmoplastic small round cell tumor. Oncology. 2018 Mar;23(3):360–366.

 Google Scholar

de Sanctis R, Bertuzzi A, Bisogno G, et al. Imatinib mesylate in desmoplastic small round cell tumors. Future Oncol. 2017 Jun;13(14):1233–1237.

 PubMedGoogle Scholar

Tap WD, Demetri G, Barnette P, et al. Phase II study of ganitumab, a fully human anti–type-1 insulin-like growth factor receptor antibody, in patients with metastatic Ewing family tumors or desmoplastic small round cell tumors. J Clin Oncol. 2012 May 20;30(15):1849–1856.

 PubMed Web of Science ®Google Scholar

Italiano A, Kind M, Cioffi A, et al. Clinical activity of sunitinib in patients with advanced desmoplastic round cell tumor: a case series. Target Oncol. 2013 Sep 6;8(3):211–213.

 PubMed Web of Science ®Google Scholar

Emambux S, Kind M, le Loarer F, et al. Clinical activity of eribulin in advanced desmoplastic small round-cell tumor. Anticancer Drugs. 2017 Oct;28(9):1053–1055.

 PubMedGoogle Scholar

Chen HM, Feng G. Use of anlotinib in intra-abdominal desmoplastic small round cell tumors: a case report and literature review. Onco Targets Ther. 2018;12:57–61.

 PubMedGoogle Scholar

Cheng K, Liu X, Chen Y, et al. Response to chemotherapy combined with anlotinib plus anlotinib maintenance in intra-abdominal desmoplastic small round cell tumors (IADSRCT): a case report and literature review. BMC Gastroenterol. 2022 Aug 17;22(1):388.

 PubMedGoogle Scholar

Fine RL, Shah SS, Moulton TA, et al. Androgen and c-Kit receptors in desmoplastic small round cell tumors resistant to chemotherapy: novel targets for therapy. Cancer Chemother Pharmacol. 2007 Jan 15;59(4):429–437.

 PubMedGoogle Scholar

Slotkin EK, Mauguen A, Ortiz MV, et al. A phase I/II study of prexasertib in combination with irinotecan in patients with relapsed/refractory desmoplastic small round cell tumor and rhabdomyosarcoma. J Clin Oncol. 2022 Jun 01;40(16_suppl):11503.

 Google Scholar