Leptomeningeal disease as a presenting feature of gestational trophoblastic neoplasia: A review and recommendations for management

• Published in: Gynecologic oncology Vol. 172; pp. 47 - 53
• Main Authors: Hapuarachi, B., Tidy, J.A., Romanowski, C., Singh, K., Gillett, S., Ireson, J., Winter, M.C.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-05-2023
• Subjects: Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Chemotherapy; Cisplatin; CNS disease; Cyclophosphamide; Dactinomycin; Etoposide; Female; Gestational Trophoblastic Disease - drug therapy; Gestational Trophoblastic Disease - therapy; Gestational trophoblastic neoplasia; Humans; Leptomeningeal disease; Methotrexate; Paclitaxel - therapeutic use; Placenta - pathology; Pregnancy; Quality of Life; Retrospective Studies; Vincristine; Leptomeningeal disease; Chemotherapy; CNS disease; Gestational trophoblastic neoplasia
• ISSN: 0090-8258; 1095-6859
• DOI: 10.1016/j.ygyno.2023.03.007

Gestational Trophoblastic Neoplasia (GTN) is a rare group of malignant placental-related tumours requiring systemic anti-cancer treatment. Leptomeningeal disease (LMD) related to GTN is not well reported with no consensus in optimal treatment. We offer recommendations for management of these patients.
We discuss five patients with GTN who presented with features of LMD and were diagnosed with gadolinium-enhanced MRI brain, all of whom received low dose induction etoposide-cisplatin (EP) followed by either EP-etoposide, methotrexate (CNS) and actinomycin-D (EMA) or EMA(CNS)-cyclophosphamide and vincristine (CO).
Four out of the five patients additionally received intrathecal methotrexate. Four patients had complete hCG response to first line multi-agent chemotherapy, one patient required second line paclitaxel, cisplatin alternating with paclitaxel, etoposide (TP/TE), where paclitaxel was substituted with nab-paclitaxel due to anaphylaxis, followed by hysterectomy. One of the four initial complete hCG responders relapsed in the lung requiring further systemic treatment with subsequent lobectomy. Patient reported outcomes indicate persistent neurological symptoms are mild and do not affect functionality and quality of life.
With a follow-up range of 2–6 years, all five patients remain cured demonstrating excellent survival outcomes with the avoidance of whole-brain radiotherapy in all cases.
•Leptomeningeal disease (LMD) due to GTN is rarely reported, and consequently there is no consensus for optimal management.•Diagnostic workup should include gadolinium-enhanced MRI brain +/− completion MRI spine.•We recommend low-dose induction EP followed by platinum-based chemotherapy and intrathecal MTX.•Whole brain radiotherapy can be avoided and should not be recommended.•We report a 100% cure rate of all five of our patients with GTN with LMD.