hemophagocytic lymphohistiocytosis (HLH)
Conditions
Interventions
Single arm group:All enrolled patients will receive RUX monotherapy as frontline therapy. Patients who have a favorable response will remain on RUX monotherapy for a total of 4-weeks. Patients with un
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
0.3 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. Patients aged > 3 months and = 38?) and are assessed to have active HLH at enrollment are eligible to participate; 5. Patient, parent, or legal authorized representative (LAR) must provide informed consent.
Exclusion criteria
Exclusion criteria: Patients who had any one of the following were ineligible: 1. Patients with known active malignancy or autoimmune diseases (e.g., systemic lupus erythematosus, dermatomyositis, autoimmune lymphoproliferative syndrome); 2. Patients with active mycobacteria, histoplasma capsulatumparasitic, leishmania, fungal infection or other uncontrolled infection; 3. Patient has severe CNS involvement presenting with obvious brain MRI abnormalities or neurological dysfunctions including convulsions, disturbance of consciousness, paralysis; 4. Patient has severe organ dysfunction, including serious renal dysfunction (creatinine clearance 20, cardiorespiratory failure requiring any extracorporeal life support; 5. Patient with organ active bleeding (including gastrointestinal hemorrhage, alveolar hemorrhage, intracranial hemorrhage, etc.); 6. History of severe allergic, anaphylactic shock, or other hypersensitivity reactions to chemicals; 7. Patient is taking another investigational agent; 8. Patients are unable to comply.
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Overall Response (ORR) rate;the overall response rate (ORR) at the end of frontline therapy with RUX monotherapy, including complete response and partial response;Duration of responses with RUX monotherapy;Correlation between RUX responses and baseline characteristics;Dynamic changes of HLH indicators during RUX monotherapy;Complete response rate with intensive therapy;6-month event-free survival rate of RUX frontline therapy and intensive therapy, respectively;Course of death; | — |
Primary
| Measure | Time frame |
|---|---|
| the 1-year overall survival (OS) rate of the treatment protocol;Adverse events (AEs) of the treatment protocol;Complete Response (CR) rate; | — |
Countries
China
Contacts
Public ContactTian-You Wang, Rui Zhang
Beijing Children’s Hospital
Outcome results
None listed