New hope for HLH patients: targeted drug tested in clinical trial

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Researchers evaluate patients with newly diagnosed or refractory/relapsed HLH disease. 2. Definite diagnosis of HLH: molecular diagnosis consistent with pHLH, or at least 5 of the 8 criteria in the HLH-2004 diagnostic criteria. 3. Patients who are currently unsuitable or unable to undergo allo-HSCT. 4. Researchers estimate that the expected survival period exceeds one month. 5. Patients must be at least 14 years of age and no older than 70 years of age, with no gender restrictions. 6. Before the study began, TB was ≤10 times the upper limit of normal; Cr was ≤1.5 times the normal value. 7. Serum HIV antigen or antibody negative。 8. HCV antibody negative, or HCV antibody positive but HCV RNA negative. 9. .HBsAg and HBcAb are both negative. If either of the above is positive, peripheral blood hepatitis B virus DNA titer testing is required, with a titer of less than 1×10³ copies/ml. 10. Echocardiography showed LVEF ≥ 50%. 11. Women of childbearing age must be confirmed as not pregnant by a pregnancy test and must be willing to use effective contraception during the study period and for at least 12 months after the last dose; all male participants must use contraception during the study period and for at least 3 months after the last dose. Exclusion Criteria: 1. Heart function above grade II(including II) (NYHA). 2. Pregnancy or lactating Women and reproductive-age patients who refused to use appropriate contraceptive measures during this trial. 3. Individuals who are allergic to GO or have a severe allergic constitution. 4. Active bleeding of the internal organs. 5. uncontrollable infection. 6. Severe mental illness. 7. History of non-melanoma skin cancer. 8. Unable to comply during the trial and/or follow-up phase. Participate in other clinical research at the same time.