Clinical Outcomes of Patients with Rare and Heavily Pretreated Solid Tumors Treated according to the Results of Tumor Molecular Profiling

Table 2

Tumor type and clinician and MP-guided therapy recommendation, MP results, and clinical outcomes: rare tumor group.

Tumor location	Prior lines	Next best treatment: clinician-defined	Next best treatment: MP-defined	Clinical observations	Biomarkers with/without benefit	Chemotherapy versus biological/targeted therapy	PFS ratio
Ethmoid sinus	1		Pemetrexed	CB	7/7	3 versus 6	1.35
Adrenal cortex (adenocarcinoma)	2		FOLFIRI	CB	6/15	16 versus 0	3.33
Thyroid (anaplastic)	1	Nil	FOLFIRI	CB	7/3	12 versus 0	0.76
Fibrosarcoma	0		Anthracyclines + dacarbazine (MAID)	CB	7/16	16 versus 0	NA
Astroblastoma	2	Temozolomide	Temozolomide	CB	4/9	5 versus 0	NA
Pseudopapillary mucinous neoplasm	1	FOLFIRI	Cisplatin + 5-FU	CB	9/9	22 versus 0	4.25
Endometrial stromal sarcoma	1		Tamoxifen (also platinum therapy, 5-FU, irinotecan, gemcitabine, and nab-paclitaxel)	P	9/9	22 versus 0	NA
Medullary thyroid	0	Nil	Carboplatin + nab-paclitaxel	NED	3/19	7 versus 0	NA
Adrenal cortical carcinoma	1	Nil	Cyclophosphamide + doxorubicin + cisplatin	P	11/1	17 versus 0	NA
Liver leiomyosarcoma	2	Pazopanib	Gemcitabine + nab-paclitaxel then nab-paclitaxel only	CB	12/4	18 versus 0	3.01
Uterine carcinosarcoma	2	Cisplatin + liposomal doxorubicin	Cisplatin + liposomal doxorubicin	P	5/11	6 versus 0	0.19
Fibrolamellar hepatocellular carcinoma	1	Sorafenib	FOLFIRI	CB	8/7	9 versus 0	1.75
Carcinoma with unknown primary (mediastinum)	2		Cisplatin + nab-paclitaxel	CB	8/13	6 versus 0	1.43
Adenoid cystic carcinoma of the maxilla (metastatic)	1		FOLFIRI	CB	8/8	9 versus 0	NA
Carcinoma with unknown primary (sacrum)	2		Cisplatin + gemcitabine	CB	3/12	7 versus 0	NA
Uterine leiomyosarcoma	3	Pazopanib	Gemcitabine + nab-paclitaxel	CB	13/2	21 versus 0	NA
Totals				CB = 11; = 3; other = 2	120/145	196 versus 6

CB = clinical benefit (QOL/PS/general symptoms/weight and imaging results improved or stable); NA = not available; NED = no evaluable disease; P = progression (QOL/PS/general symptoms/weight and imaging results declined or deteriorated).
Ratio of PFS with therapy suggested by MP versus that with prior line of therapy.
FOLFIRI, irinotecan + 5-FU + folinic acid; MAID, mesna + doxorubicin + ifosfamide + dacarbazine; PFS, progression-free survival; PS, performance status; QOL, quality of life.