Castleman disease (CD) is a rare disorder of the hematological system, included in China’s first catalog of rare diseases. Professor Li Jian’s team at PUMCH has long been dedicated to clinical research of this disease. The team took the lead in establishing China Castleman Disease Network (CCDN), which faciliated multi-disciplinary cooperation within a hospital and cooperation between hospitals. Recently, the team has published a series of papers in distinguished academic journals such as “The Lancet Regional Health - Western Pacific”, “Blood” and “Chest”. The publications outlined the diagnosis and treatment of CD in China over the past 20 years, reported the effectiveness of the BCD regimen (bortezomib, cyclophosphamide, and dexamethasone) in the first-line treatment of iMCD (idiopathic multicentric CD) patients, and portrayed the characteristics and evolution of pulmonary involvement in iMCD-not otherwise specified (iMCD-NOS) patients. The team’s series of research outcomes have steadily advanced the clinical research process of CD and improved clinicians’ understanding of this rare disease.

This study enrolled 1,634 CD patients over the past 21 years from 2000 to 2021 at 40 Chinese institutions to describe distribution, treatment options, and prognostic factors of the disease.

▲Figure 1. Evolution of first-line treatment of iMCD in China

This was the largest retrospective study of CD carried out to date, which implemented the CDCN diagnostic criteria for CD and delineated CD subtypes. For the first time, the concept of severe iMCD proposed by the CDCN was validated in this study. Severe iMCD patients could benefit from early identification treated with enhanced therapies. The study was highly valued and published as a commentary by “The Lancet Regional Health – Western Pacific”.

Interleukin-6 (IL-6)-targeted therapy is the first-line treatment of choice for iMCD patients. However, the IL-6-targeted therapy is not effective or affordable for all patients (overall response rate is 34% in randomized controlled studies). Professor Li Jian’s team has been working hard to develop affordable, convenient and effective treatment options for the Chinese population that use alternative targets other than IL-6. This study demonstrated the efficacy and safety of the BCD regimen as first-line treatment of iMCD. More importantly, the study showed that the regimen was effective in patients with severe iMCD and iMCD-TAFRO and even, to some extent, overcame the adverse effects on prognosis for patients with these subtypes.

▲Figure 2. Efficacy of the BCD regimen as the first-line therapy for iMCD

Pulmonary involvement is an important complication of iMCD. The teams of Prof. Zhang Weihong from the Department of Radiology and of Prof. Li Jian from the Department of Hematology collaborated on a study that tackled this problem. The study enrolled 58 patients with iMCD-NOS and described the imaging manifestations, natural evolution pattern, and possible reversibility after treatment for this specific clinical subtype of pulmonary involvement.

The results showed that iMCD-NOS pulmonary involvement manifestations included nodules (96.6%), cysts (65.5%), consolidation (22.4%), interstitial thickening (50.0% to 87.9%), and ground-glass opacities (55.2%). The investigators also found that all pulmonary lesions other than cysts improved after effective systemic therapy.

▲Figure 3. CT imagings of the three subgroups (A and B: nodules; C and D: cysts; E and F: consolidation)