OBJECTIVES: For ultra rare diseases independent case reports provide useful real-world evidence on disease burden and treatment trends. The objective of this review was to understand incidence and treatment trends for Veno-Occlusive Disease (VOD) using latest published case reports.

METHODS:

PubMed, Medline and NOVEL-VOD databases were searched for English language publications dated January 1, 2018 to December 31, 2022. Case reports were selected for inclusion if publications reported incidence or treatment for VOD (also called SOS-Sinusoidal Obstruction Syndrome). Selected publications were tagged as epidemiology, treatment trends, defibortide treated, pulmonary or hepatic VOD. Each category of case reports were reviewed to develop trends, insights and learnings for latest trends in VOD.

RESULTS:

A total of 347 full text records on VOD/SOS were identified, of which 46 included case reports. The individual case reports and case services cumulatively reported on VOD incidence or treatment in 99 patients. In 30 patients, the incidence of VOD was induced due to chemotherapies such as pyrrolizidine alkaloids, thioguanine and oxaliplatin. Defibrotide was used in 12 patients. Pulmonary VOD was reported in 14 case reports with most mentioning challenges in diagnosis; hepatic VOD was reported in 13 publications. In 11 case reports, severe or very severe forms of VOD were reported due to chemotherapy and/or conditioning regimens for transplant. Treatment related case reports mention early diagnosis and timely treatment of VOD led to improved outcomes.

CONCLUSIONS: Latest case reports provide potentially useful real-world evidence on incidence and treatment trends for rare disease such as VOD.