Longitudinal, natural history study reveals the disease burden of idiopathic multicentric Castleman disease Journal Article
| Authors: | Bustamante, M. S.; Pierson, S. K.; Ren, Y.; Bagg, A.; Brandstadter, J. D.; Srkalovic, G.; Mango, N.; Alapat, D.; Lechowicz, M. J.; Li, H.; van Rhee, F.; Lim, M. S.; Fajgenbaum, D. C. |
| Article Title: | Longitudinal, natural history study reveals the disease burden of idiopathic multicentric Castleman disease |
| Abstract: | Idiopathic multicentric Castleman disease (iMCD) is a rare hematologic disorder with heterogeneous presentations ranging from moderate constitutional symptoms to life-threatening multiorgan system involvement. There are vastly different clinical subtypes, with some patients demonstrating thrombocytopenia, anasarca, fever/elevated C-reactive protein, reticulin fibrosis/renal failure, and organomegaly (TAFRO) and others having milder/more moderate symptoms with potential for severe disease (not otherwise specified, NOS). Due to its rarity and heterogeneity, the natural history and long-term burden of iMCD are poorly understood. We investigated real-world medical data from ACCELERATE, a large natural history registry of patients with Castleman disease, to better characterize the long-term disease burden experienced by these patients. We found that iMCD-TAFRO patients face a significant hospitalization burden, requiring more time in the hospital than iMCD-NOS patients during the year surrounding diagnosis (median [interquartile range]: 36 [18-61] days vs. 0 [0-4] days; P<0.001). In addition, we found life-sustaining interventions, such as mechanical ventilation (17%) and dialysis (27%), were required among iMCD patients, predominantly those with iMCD-TAFRO. iMCD-NOS patients, however, spent a significantly greater proportion of time following disease onset in a state of disease flare (median 52.3% vs. 18.9%; P=0.004). Lastly, we observed severe iMCD-related morbidities, such as acute renal failure, sepsis and pneumonia, among others, arising after iMCD diagnosis, impairing the patients’ quality of life. These data demonstrate a substantial disease burden experienced by iMCD patients and emphasize the importance of ongoing research into iMCD to aid disease control. ©2024 Ferrata Storti Foundation Published under a CC BY-NC license. |
| Keywords: | adult; clinical article; controlled study; aged; middle aged; major clinical study; prednisone; histopathology; case report; hypertension; rituximab; c reactive protein; quality of life; thrombocytopenia; morbidity; cohort analysis; obesity; bendamustine; cyclophosphamide; pathology; fever; pneumonia; hypoalbuminemia; social support; depression; hospitalization; disease severity; myelodysplastic syndrome; register; registries; longitudinal studies; bone marrow biopsy; heart failure; hepatomegaly; splenomegaly; diagnosis; sepsis; health care system; human immunodeficiency virus; epidemiology; corticosteroid; drug therapy; observational study; disease control; gastroesophageal reflux; tacrolimus; erythrocyte transfusion; cost of illness; cholecystitis; disease exacerbation; lymphadenopathy; chronic kidney failure; thrombocytosis; longitudinal study; thrombocyte transfusion; fluid retention; human experiment; hepatosplenomegaly; disease activity; complication; reticulin; microangiopathy; angiofollicular lymph node hyperplasia; anasarca; tocilizumab; disease burden; humans; human; male; female; article; siltuximab; castleman disease; multi-centric castleman's disease; multicentric castleman disease |
| Journal Title: | Haematologica |
| Volume: | 109 |
| Issue: | 7 |
| ISSN: | 0390-6078 |
| Publisher: | Ferrata Storti Foundation |
| Date Published: | 2024-07-01 |
| Start Page: | 2196 |
| End Page: | 2209 |
| Language: | English |
| DOI: | 10.3324/haematol.2023.283603 |
| PUBMED: | 38205523 |
| PROVIDER: | scopus |
| PMCID: | PMC11217716 |
| DOI/URL: | |
| Notes: | Article -- Erratum issued, see DOI: 10.3324/haematol.2024.286500 -- Source: Scopus |
