Clinical Trial: Pilot Study of Etoposide-based Therapy and Hematopoietic Cell Transplantation for Hemophagocytic Lymphohistiocytosis

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Pilot Study of First-line Immunosuppressive Therapy Combined With Etoposide and Allogeneic Hematopoietic Cell Transplantation in Refractory/Reactivated Cases for Hemophagocytic L

Brief Summary: The investigators would like to propose a pilot study evaluating the efficacy of etoposide combined with immunosuppressive agents for adult secondary hemophagocytic lymphohistiocytosis (HLH), in order to prove out whether the modification of previous HLH-94 or HLH-2004 protocol for childhood patients can improve the outcome and decrease the toxicities. The results of this pilot study will be a base of a more-improved phase-2 protocol.

Detailed Summary: The treatment of HLH in adult patients has not been determined yet. Actually, we adopted the treatment protocol HLH2004, which was developed for pediatric HLH patients. The HLH2004 protocol, which is a potent and successful treatment for HLH, has shown some limitations in the treatment of adult HLH. First, the dose of etoposide is somewhat high for adult patients to tolerate. Second, the high incidence of opportunistic infection such as fungal, bacterial, and viral has threatened the patients. Third, more aggressive and intensive approach to adopt allogeneic hematopoietic cell transplantation will be needed earlier in adult patients. Based on these rationales, we developed a modified protocol based on HLH2004 to pit the treatment of adult HLH patients.
Sponsor: Asan Medical Center

Current Primary Outcome: overall survival rate [ Time Frame: 1-year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Complete response rate [ Time Frame: 28 days ]
  • Complete response rate [ Time Frame: 56 days ]
  • Complete response rate [ Time Frame: 3 months ]
  • Partial response rate [ Time Frame: 28 days ]
  • Partial response rate [ Time Frame: 56 days ]
  • Partial response rate [ Time Frame: 3 months ]
  • reactivation-free survival rate [ Time Frame: 3 months ]
  • reactivation-free survival rate [ Time Frame: 6 months ]
  • overall survival rate [ Time Frame: 3 months ]
  • overall survival rate [ Time Frame: 6 months ]
  • overall survival rate [ Time Frame: 1 year ]
  • treatment-related mortality rate [ Time Frame: 1 year ]


Original Secondary Outcome: Same as current

Information By: Asan Medical Center

Dates:
Date Received: February 29, 2012
Date Started: March 2012
Date Completion: February 2019
Last Updated: May 22, 2016
Last Verified: May 2016