Table 1.

Current summary of reported patients with GTN treated with CPI

Study	Type of study	Patients, n	Diagnosis	Clinical setting	Therapeutic agent	Outcome	Grade 3 or 4 toxicity
Ghorani et al. [20] (2017)	Clinical case report	4	2 choriocarcinoma	Multidrug resistant disease	Pembrolizumab	3 CR, 1 PD	No
			1 PSTT
			1 PSTT/ETT mixed type
Huang et al. [25] (2017)	Clinical case report	1	Choriocarcinoma	Multidrug resistant disease	Pembrolizumab	CR	Grade 3 transaminitis
Choi et al. [26] (2019)	Clinical case report	2	1 PSTT	Multidrug resistant disease	Pembrolizumab	CR	No
			1 ETT
			Both tumours dMMR
Cheng et al. [27] (2020)	Clinical case report	8	7 choriocarcinoma	Multidrug resistant disease	Unknown (anti-PD-1)	4 CR	No
						4 relapsed, different systemic treatment, no follow-up information
			1 ETT (abstract only, paper in Chinese)
Goldfarb et al. [28] (2020)	Clinical case report	1	Choriocarcinoma	Multidrug resistant disease	Pembrolizumab	CR but relapse after 22 months, pembro reinstalled, no follow-up information	Grade 3 neuropathy
Clair et al. [29] (2020)	Clinical case report	1	Choriocarcinoma	Multidrug resistant disease	Pembrolizumab	CR	Unknown
Bell et al. [30] (2021)	Clinical case report	1	ETT	Multidrug resistant disease	Pembrolizumab	CR	Unknown
Paspalj et al. [31] (2021)	Clinical case report	1	Choriocarcinoma	Multidrug resistant disease	Pembrolizumab	CR	No
Pisani et al. [32] (2021)	Clinical case report	1	ETT	Multidrug resistant disease	Pembrolizumab	CR	No
Cheng et al. [23] (2021)	Prospective phase II trial	20	19 choriocarcinoma	High-risk (FIGO>7) chemotherapy refractory or relapsed GTN	Camrelizumab and Apatinib	10 CR	12 patients grade 3 toxicities
						10 PD and received multi-modality treatment
			1 PSTT
You et al. [21] (2020)	Prospective phase II trial	15	Low-risk GTN	GTN progressive after single-agent chemotherapy	Avelumab	8 CR (and 1 healthy pregnancy post treatment)	No
						7 PD, reached CR after Act-D or multi-modality treatment
Posnaszek et al. [33] (2021)	Clinical case report	1	PSTT	Uterine tumour, patient declined surgery and chemotherapy	Pembrolizumab	CR after 3 cycles (and healthy pregnancy after treatment)	Unknown
You et al. [24] (2023)	Prospective phase II trial	7	High-risk GTN	Multidrug resistant disease	Avelumab	1 CR	No
						6 PD, study stopped for futility
Liu et al. [22] (2022)	Clinical case report	4	1 GTN not specified	Multidrug resistant disease	Toripalimab combined with chemotherapy	4 CR	No
			3 choriocarcinoma
Wang et al. [34] (2023)	Retrospective cohort study	66	59 choriocarcinoma	Chemo-refractory or relapsed GTN (all FIGO score >7)	Camrelizumab (37)	CR 46	25 patients2
					Sintilimab (8)
					Toripalimab (11)	PR 6
					Pembrolizumab (10)1	No response 14
			7 PSTT/ETT

TKI, tyrosine kinase inhibitor.

¹In 31 patients, CPI was combined with different schedules of chemotherapy; 20 patients received apatinib (TKI) in addition to anti-PD-1 therapy.

²25 (of 66) patients with grade 3–4 toxicity reported received; anti-PD1 only: 10 patients (out of 35); anti-PD-1 plus chemotherapy: 15 (out of 31).