A retrospective survey of patients who discontinued participation in the JPLSG HLH-2004 clinical trial

Although clinical trials have reported an improvement in the prognosis of hemophagocytic lymphohistiocytosis (HLH), current treatment outcomes are unsatisfactory, especially in severe cases. Most clinical trial patients with severe disease discontinue participation due to complications associated wi...

Full description

Saved in:
Bibliographic Details
Published in:International journal of hematology 2022-09, Vol.116 (3), p.434-441
Main Authors: Ono, Rintaro, Sakamoto, Kenichi, Doi, Takehiko, Yanagisawa, Ryu, Tamura, Akihiro, Hashimoto, Hiroya, Kanegane, Hirokazu, Ishii, Eiichi, Nakazawa, Yozo, Shioda, Yoko
Format: Article
Language:English
Subjects:
Clinical trials
Complications
Encephalopathy
Health services
Hematology
Histiocytosis
Lymphocytosis
Medical prognosis
Medicine
Medicine & Public Health
Oncology
Original Article
Patients
Surveys
Survival
Toxicity
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Although clinical trials have reported an improvement in the prognosis of hemophagocytic lymphohistiocytosis (HLH), current treatment outcomes are unsatisfactory, especially in severe cases. Most clinical trial patients with severe disease discontinue participation due to complications associated with HLH or treatment-related toxicity. A retrospective survey of patients who discontinued participation in the JPLSG HLH-2004 clinical trial was conducted to review the detailed course of these cases to optimize HLH treatment and supportive care. Findings in these patients were compared with those of 45 patients who completed the protocol treatment. The 3 year overall survival rate of patients who completed treatment was 86.7%, versus 50.7% for those who did not complete treatment. Incidence of serious adverse events, such as infections, coagulopathy, and posterior reversible encephalopathy syndrome, during the initial 8 weeks of treatment was much higher in patients who did not complete treatment than in patients who completed treatment. To improve overall outcomes of patients with HLH, it is important to not only optimize HLH-directed therapy but also provide appropriate supportive care.
ISSN:0925-5710
1865-3774
DOI:10.1007/s12185-022-03357-1