Desmoplastic Small Round Blue Cell Tumor: A Review of Treatment and Potential Therapeutic Genomic Alterations

Table 1

Summary of patients’ characteristics, treatments, and outcome in DSRCT.

Study	Number of patients	Type of study	Age range	Chemo	Cytoxan dose	Response	Survival	Additional Rx	Comment
Kushner et al. [55]	10 (untreated patients)	Prospective	7–22 (median, 14 yrs)	P6	4.2 g/m^2 over 2 days	PR 70%; CR 20% (no path CR)	Median OS 19 mo (22 for 7 pts in CR). 5 remained in CR at 38 mo	40% RT; 30% BMT; 30% ABMT^#	1 tumor-related Budd-Chiari death. Carboplatin/thiotepa for myeloablative transplant
Hayes-Jordan et al. [29]	24	Retrospective	8–43 (median, 12 yrs)	P6	4.2 g/m^2 over 2 days	RR not reported. Complete resection to less than 1.0 cm tumor size was achieved in all 8 patients who underwent HIPEC	3 yr OS: HIPEC + Sx = 71%; chemo/RT = 26%; Sx alone = 62%	HIPEC cisplatin	HIPEC only used in 5–25 yr age group. Thoracic metastasis suggested poor prognosis
Lal et al. [56] (MSKCC)	66	Retrospective	7–58 (median, 19 yrs)	P6	4.2 g/m^2 over 2 days	Not reported	3 yr OS 44%; 5 yr OS 15%; 3 yr OS 58% with GTR	CPT-11, topotecan, carboplatin, cisplatin were added in selected patients	In 71%, greater than 90% tumor resection was possible. 71% underwent Rx with P6 regimen
Farhat et al. [63]	5	Retrospective	16–26 (median 22 yrs)	PA(E)VP	900 mg/m^2 over 3 days	4 stable disease 1 CR	Mean survival 24 mo	ABMT (carboplatin, 800 mg/m^2; etoposide, 1200 mg/m^2; and ifosfamide, 6 g/m^2) in 1 patient	Chemotherapy was given adjuvantly. 1 CR was reported to have tunica vaginalis (primary)
Pinnix et al. [58]	8	Retrospective	5–20 (median, 11 yrs)	P6	4.2 g/m^2 over 2 days	5/8 had complete resection; 2/8 had near complete (>90%) resection	At 30 mo, three patients died of PD, four were alive with active disease, and one was in CR	7/8 patients had HIPEC	25% had extra-abdominal metastasis. Mean time to IMRT failure 6.6 mo. 70–80% Gr 2 GI toxicity. Limited Gr ½ hematological toxicity mostly anemia
Goodman et al. [57] (MSKCC)	21	Retrospective	8–34 (median, 16.5 yrs)	P6	4.2 g/m^2 over 2 days	Not reported. Maximal debulking in all but 1 patient	3 yr OS 48%; 3 yr RFS 14%; median OS 32 mo	Cisplatin, carboplatin, topotecan, irinotecan, and vinorelbine were also used. 30 Gy WA-XRT	Grade 4 thrombocytopenia, leukopenia, and anemia in 76%, 29%, and 33%, respectively. Bowel obstruction in 33%
Wong et al. [98]	41	Retrospective	16–45 (median, 27 yrs)	Vincristine + ifosfamide + doxorubicin + etoposide (VIDE) in a 1/3rd of 1st line Rx	Ifos 3 g/m^2 over 3 days	Not reported	3 yr OS 27%; 5 yr OS 16%	6/41 received XRT	VIDE chemotherapy appeared to confer the longest TTP (median, 14.6 months)
Aguilera et al. [73]	1 (5 yr old, only outpatient regimen)	Case report	5 yrs	VIDE (vincristine (1.5 mg/m^2), dexrazoxane/doxorubicin (750/75 mg/m^2), and etoposide (150 mg/m^2))	Ifos 3 g/m^2 over 3 days (outpatient)	R0 resection with microscopic residual disease	Relapse at 18 months. Alive at 2 yrs after Dx	HIPEC cisplatin 100 mg/m^2 and aggressive tumor debulking. Followed by Temodar/irinotecan maintenance x12 followed by IMRT (30 Gy)	Ifosfamide infusions were done at home with bag changes by home health nursing. Retroperitoneal relapse treated with IMRT with bevacizumab (5 mg/kg) and 2 perihepatic metastases with radiofrequency ablation/cryoablation followed by chronic outpatient maintenance chemotherapy (valproic acid, cyclophosphamide, and rapamycin)

^#ABMT = autologous myeloablative transplant. GTR = gross tumor resection. There was no statistical difference in estimated OS for those who received debulking surgery compared with HIPEC, in those who did not receive HIPEC. There were no survivors greater than 3 years.