Langerhans cell histological hyperplasia (LCH) is a histocellular disease formerly known as "histocellular hyperplasia X". Its characteristics are clonal proliferation of histocytes and excessive accumulation of pathological Langerhans cells. The most common involved organs include: bone (80%), skin (33%), pituitary (25%), etc. When dangerous organs are involved, such as the blood system, liver, and spleen, the patient is at risk of dying, has a poor response to treatment, and has a poor prognosis.
Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histological hyperplasia, formerly known as "lipid granuloma". ECD lesions can involve in all systems throughout the body. The most common affected systems include: bones, cardiovascular, respiratory, central nervous system , etc. General symptoms include fever, fatigue, night sweats, weight loss, etc.
Langerhans cell histiocytic hyperplasia (LCH) and Erdheim-Chester disease are caused by mutations in the MAPK pathway ( is most commonly BRAF V600E) in myeloid dendritic cells, thus leading to both overlap and their own unique manifestations.
Due to the rareness of the above two diseases, there are certain challenges in their diagnosis and treatment. MEK inhibitor and BRAF inhibitor are important progress in recent years in application.
Free Clinical Project
MEK Inhibitor FCN-159
Trial Title: A multi-center, open, single-arm phase II study evaluating the effectiveness, safety and pharmacokinetic characteristics of FCN-159 in patients with histocellular tumors.
Test type: Single-arm test
Indications: Langerhans cell histological hyperplasia and Erdheim-Chester disease and other histocellular tumors (first-line and above)
Registration number: CTR20221069
Research Center: Beijing, Zhengzhou, Henan, Changsha, Hunan, Shanghai, Chengdu, Sichuan, Hangzhou, Zhejiang, Hefei, Hebei, Shijiazhuang, Shenyang, Liaoning,
Main entry standards:
1, age 16 years old; expected survival time at least 3 months; ECOG physical strength score 0-2 points. Patients with Langerhans cell histological hyperplasia (LCH), Erdheim-Chester disease (ECD) and other histological tumors were diagnosed with
and histology .
3, can provide sufficient number of tissue pathological sections or tumor tissue samples for central pathological review and central laboratory testing of the following biomarker : ERBB3, RAF-1, BRAF, ARAF, HRAS, KRAS, NRAS, MEK (MAP2K1 and MAP2K2) and other MEK upstream genes .
4, LCH patients require lesions to involve multiple system (greater than one system) or to involve single system multi-lesion lesions (greater than one lesion).
5. There are lesions that can be evaluated based on the PET response criteria (PRC). Patients with LCH, ECD or other histocyte tumors who are initially treated or relapsed or refractory to can be included.
7. Sufficient liver and renal function: aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤2.5×Upper limit of normal value (ULN), if liver invasion occurs, ≤5×ULN; serum total bilirubin ≤1.5×ULN, if liver invasion occurs, ≤5.0×ULN, if liver invasion occurs, ≤5.0×ULN; alkaline phosphatase (AKP) ≤2.5×ULN, if liver invasion occurs, ≤10×ULN; serum creatinine ≤1.5×ULN, if serum creatinine >1.5×ULN, creatinine clearance (CCr) ≥50 mL/min; albumin ≥3g/dL.
8, do not interstitial pneumonia patients, including radiopneumonia with clinical significance. Excluding interstitial pneumonia caused by primary lung involvement.
9. Previous or current retinal venous occlusion (RVO), retinal pigment epithelial dissection (RPED), glaucoma and other meaningful abnormalities ophthalmic examinations cannot participate in .
10. Patients who have received one of the following treatments cannot participate in :
a. After 4 weeks before the drug treatment is started, they have received chemotherapy, targeted therapy, immunotherapy, , biological therapy, experimental therapy or herbal anti-tumor treatment for histocellular tumors within 4 weeks or 5 half-life (whichever is longer).
b. I received strong CYP3A4, CYP2C8, CYP2C9 inhibitors or inducers within 14 days before starting the study drug treatment, except for external use in skin.
c. growth factor that promotes the number or function of platelet or leukocyte within 7 days before starting the study drug treatment.
d. Within 4 weeks before the start of the study of drug treatment, I received radiation therapy and surgical treatment.
e. Patients who participated in other interventional clinical trials within 4 weeks before the start of the study of drug treatment.
f. The MEK1/2 inhibitor (smetinib, bimetinib, etc.) has been used in the past.
g. Large-dose chemotherapy and stem cell transplant rescue (autologous stem cell transplantation) or allogeneic stem cell transplantation treatment were performed within 90 days before enrollment. Patients who receive anti-graft-versus-host disease (GVHD) drugs such as cyclosporine, tacrolimus or other drugs for preventing GVHD after bone marrow transplantation cannot participate in this trial.
h. For patients with brain tumors (intracranial mass), anticoagulants were used within 7 days before starting the study drug treatment.
i. Corticosteroid treatment is allowed within one month before enrollment, but must be stopped 14 days before initiation of study drug treatment. Before enrollment, patients with brain lesions receiving corticosteroids for brain edema and must maintain a stable dose within 14 days.
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