San Paulo, Brazil

Establishment of an Early Warning Screening System for Hemophagocytic Lymphohistiocytosis a Multi-center, Prospective Study

Mirdametinib in Histiocytic Disorders

Langerhans cell histiocytosis (LCH) is a rare blood disorder. Though affecting all ages, LCH occurs more often in children, with an increased incidence in children less than 1 year of age. The disease presents in various ways, with most children suffering bony lesions, and skin rashes. In some patients, LCH affects vital organs such as liver, spleen, bone marrow, and the central nervous system. This group of patients are at significant risk of serious illness and death and are thus said to have risk-organ-positive (RO+) LCH. Current treatments for LCH consist of chemotherapy combined with other medications. However, many patients, especially those with RO+ disease, do not respond to therapy. Of the patients that do respond, many suffer progression of disease after an initial response to therapy, or recurrence of disease after completion of therapy. The purpose of this study is to see if treatment with mirdametinib in patients with LCH or other histiocytic disorders will be better than current treatments and with fewer side effects.

Subtypes and Prognostic Factors in Erdheim-Chester Disease

GWAS and EWAS in Patients With Erdheim-Chester Disease

A Randomized Controlled Study of High-dose Cyclophosphamide Induction Therapy in Adult Patients With HLH

Lenalidomide and Dexamethasone for Rosai-Dorfman Disease

Supportive Care Needs of Caregivers of People With Erdheim-Chester Disease and Other Histiocytic Diseases

Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease and BRAF Mutation

PRIMARY OBJECTIVES: I. To study the efficacy and safety of dabrafenib and trametinib as combination therapy in patients with BRAFV600E positive Erdheim Chester disease. II. To determine the clinical response rate to dabrafenib and trametinib combination therapy in patients with BRAFV600E positive Erdheim Chester disease. SECONDARY OBJECTIVES: I. To determine time response, progression free survival and overall survival. II. To assess disease resistance to this combination therapy. TERTIARY OBJECTIVES: I. To monitor the degree of histiocytic infiltration-fibrosis progression, stability and regression under combination therapy using fludeoxyglucose F 18 (FDG)-positron emission tomography (PET) scan, magnetic resonance imaging (MRI) scans, computed tomography (CT) scans and technetium (T)-99m bone scans. II. To monitor serum C-reactive protein (CRP), estrogen receptor (ESR), and cytokine levels as inflammatory markers prior to and during combination therapy. III. To monitor renal function prior to and during combination therapy in order to assess for functional improvement. IV. To evaluate the level of functioning, fatigue, motor skills and ability to perform routine daily activities prior to and during therapy in order to assess for improvements in these areas as well as quality of life improvement. V. To establish duration of treatment-endpoints in patients with BRAF V600E positive Erdheim Chester disease (ECD) lesions. OUTLINE: Patients receive dabrafenib orally (PO) twice daily (BID) and trametinib PO once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 12 weeks.

Registry for Patients with Erdheim-Chester Disease and Other Histiocytoses

A Prospective Clinical Study of Ruxolitinib and Etoposide Combined With DDGP Regimen (RUE-DDGP) in Induction Therapy of T/NK Cell Lymphoma-associated Hemophagocytic Syndrome.

This is an open, one-arm, prospective clinical collaborative study. This study is aimed to observe the efficacy and safety of the new combined therapy of Ruxolitinib and Etoposide combined with DDGP regimen ( cis-Platinum, Dexamethasone, Gemcitabine and Pegaspargase) in the first-line induction therapy patients with primary central nervous system lymphoma. A total of 30 patients plan to participate in the study. The primary endpoint is objective remission rate (ORR) of hemophagocytic syndrome, and the secondary endpoints include progression-free survival (PFS) , objective remission rate (ORR) of lymphoma, overall survival (OS), and adverse events(ADR).