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Castleman disease (CD) describes a group of at least 4 disorders that share a spectrum of characteristic histopathological features but have a wide range of etiologies, presentations, treatments, and outcomes. CD includes unicentric CD (UCD) and multicentric CD (MCD), the latter of which is divided into idiopathic MCD (iMCD), human herpes virus-8 (HHV8)-associated MCD (HHV8-MCD), and polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell disorder, skin changes (POEMS)-associated MCD (POEMS-MCD). iMCD can be further subclassified into iMCD-thrombocytopenia, ascites, reticulin fibrosis, renal dysfunction, organomegaly (iMCD-TAFRO) or iMCD-not otherwise specified (iMCD-NOS). Advances in diagnosis, classification, pathogenesis, and therapy are substantial since the original description of UCD by Benjamin Castleman in 1954. The advent of effective retroviral therapy and use of rituximab in HHV8-MCD have improved outcomes in HHV8-MCD. Anti-interleukin-6-directed therapies are highly effective in many iMCD patients, but additional therapies are required for refractory cases. Much of the recent progress has been coordinated by the Castleman Disease Collaborative Network (CDCN), and further progress will be made by continued engagement of physicians, scientists, and patients. Progress can also be facilitated by encouraging patients to self-enroll in the CDCN's ACCELERATE natural history registry (#NCT02817997; www.CDCN.org/ACCELERATE).
Castleman disease (CD) describes a group of heterogeneous hematologic disorders with characteristic histopathological features. CD can present with unicentric or multicentric (MCD) regions of lymph node enlargement. Some cases of MCD are caused by human herpesvirus-8 (HHV-8), whereas others are HHV-8-negative/idiopathic (iMCD). Treatment of iMCD is challenging, and outcomes can be poor because no uniform treatment guidelines exist, few systematic studies have been conducted, and no agreed upon response criteria have been described. The purpose of this paper is to establish consensus, evidence-based treatment guidelines based on the severity of iMCD to improve outcomes. An international Working Group of 42 experts from 10 countries was convened by the Castleman Disease Collaborative Network to establish consensus guidelines for the management of iMCD based on published literature, review of treatment effectiveness for 344 cases, and expert opinion. The anti-interleukin-6 monoclonal antibody siltuximab (or tocilizumab, if siltuximab is not available) with or without corticosteroids is the preferred first-line therapy for iMCD. In the most severe cases, adjuvant combination chemotherapy is recommended. Additional agents are recommended, tailored by disease severity, as second- and third-line therapies for treatment failures. Response criteria were formulated to facilitate the evaluation of treatment failure or success. These guidelines should help treating physicians to stratify patients based on disease severity in order to select the best available therapeutic option. An international registry for patients with CD (ACCELERATE, #NCT02817997) was established in October 2016 to collect patient outcomes to increase the evidence base for selection of therapies in the future.
Castleman disease (CD) includes a group of rare and heterogeneous disorders with characteristic lymph node histopathological abnormalities. CD can occur in a single lymph node station, which is referred to as unicentric CD (UCD). CD can also involve multicentric lymphadenopathy and inflammatory symptoms (multicentric CD [MCD]). MCD includes human herpesvirus-8 (HHV-8)-associated MCD, POEMS-associated MCD, and HHV-8-/idiopathic MCD (iMCD). The first-ever diagnostic and treatment guidelines were recently developed for iMCD by an international expert consortium convened by the Castleman Disease Collaborative Network (CDCN). The focus of this report is to establish similar guidelines for the management of UCD. To this purpose, an international working group of 42 experts from 10 countries was convened to establish consensus recommendations based on review of treatment in published cases of UCD, the CDCN ACCELERATE registry, and expert opinion. Complete surgical resection is often curative and is therefore the preferred first-line therapy, if possible. The management of unresectable UCD is more challenging. Existing evidence supports that asymptomatic unresectable UCD may be observed. The anti-interleukin-6 monoclonal antibody siltuximab should be considered for unresectable UCD patients with an inflammatory syndrome. Unresectable UCD that is symptomatic as a result of compression of vital neighboring structures may be rendered amenable to resection by medical therapy (eg, rituximab, steroids), radiotherapy, or embolization. Further research is needed in UCD patients with persisting constitutional symptoms despite complete excision and normal laboratory markers. We hope that these guidelines will improve outcomes in UCD and help treating physicians decide the best therapeutic approach for their patients.
Human herpesvirus-8 (HHV-8)-negative, idiopathic multicentric Castleman disease (iMCD) is a rare and life-threatening disorder involving systemic inflammatory symptoms, polyclonal lymphoproliferation, cytopenias, and multiple organ system dysfunction caused by a cytokine storm often including interleukin-6. iMCD accounts for one third to one half of all cases of MCD and can occur in individuals of any age. Accurate diagnosis is challenging, because no standard diagnostic criteria or diagnostic biomarkers currently exist, and there is significant overlap with malignant, autoimmune, and infectious disorders. An international working group comprising 34 pediatric and adult pathology and clinical experts in iMCD and related disorders from 8 countries, including 2 physicians that are also iMCD patients, was convened to establish iMCD diagnostic criteria. The working group reviewed data from 244 cases, met twice, and refined criteria over 15 months (June 2015 to September 2016). The proposed consensus criteria require both Major Criteria (characteristic lymph node histopathology and multicentric lymphadenopathy), at least 2 of 11 Minor Criteria with at least 1 laboratory abnormality, and exclusion of infectious, malignant, and autoimmune disorders that can mimic iMCD. Characteristic histopathologic features may include a constellation of regressed or hyperplastic germinal centers, follicular dendritic cell prominence, hypervascularization, and polytypic plasmacytosis. Laboratory and clinical Minor Criteria include elevated C-reactive protein or erythrocyte sedimentation rate, anemia, thrombocytopenia or thrombocytosis, hypoalbuminemia, renal dysfunction or proteinuria, polyclonal hypergammaglobulinemia, constitutional symptoms, hepatosplenomegaly, effusions or edema, eruptive cherry hemangiomatosis or violaceous papules, and lymphocytic interstitial pneumonitis. iMCD consensus diagnostic criteria will facilitate consistent diagnosis, appropriate treatment, and collaborative research.
All Publications
2024
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Saishravan Shyamsundar, et al. "Patients with Castleman disease report mild COVID-19 symptoms and mount a humoral response to SARS-CoV-2 vaccination." Blood Neoplasia. 2024 March 21. (online ahead of print). 10.1016/j.bneo.2024.100002
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Mateo Sarmiento Bustamante, Sheila K. Pierson, et al. "Longitudinal, natural history study reveals the disease burden of idiopathic multicentric Castleman disease." Haematologica. 2024 July 1. doi: 10.3324/haematol.2023.283603
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Ayelet I Rubenstein, et al. "Immune-mediated thrombocytopenia and IL-6-mediated thrombocytosis observed in idiopathic multicentric Castleman disease." British Journal of Haematology. 2024 Jan 2 (online ahead of print). doi: 10.1111/bjh.19279
2023
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Joshua D Brandstadter, et al. "A novel cryopreservation and biobanking strategy to study lymphoid tissue stromal cells in human disease." European Journal of Immunology. 2023 Sep;53(9): e2250362. doi: 10.1002/eji.202250362
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Stefano Rodolfi, et al. "Lymphadenopathy in the rheumatology practice: a pragmatic approach." Rheumatology (Oxford). 2023 Dec 18;kead644. doi: 10.1093/rheumatology/kead644
- Mateo Sarmiento Bustamante, Saishravan Shyamsundar, et al. "Ongoing symptoms following complete surgical excision in unicentric Castleman disease." American Journal of Hematology. 2023 Nov;98(11): E334-E337. doi: 10.1002/ajh.27065
- Ania Korsunska, et al. "A model for crowdsourcing high-impact research questions for Castleman disease and other rare diseases." Orphanet Journal of Rare Disease. 2023 Apr 11;18(1): 75. doi: 10.1186/s13023-023-02678-6
- Sheila K. Pierson, et al. "Treatment consistent with idiopathic multicentric Castleman disease guidelines is associated with improved outcomes." Blood Advances. 2023 Nov 14;7(21): 6652-6664. doi: 10.1182/bloodadvances.2023010745
- Natalie A. Mango, et al. "Siltuximab administration results in spurious IL-6 elevation in peripheral blood." American Journal of Hematology. 2023 Oct 23;99(1); E15-E18. doi: 10.1002/ajh.27132
- Pedro Horna, et al. "The lymph node transcriptome of unicentric and idiopathic multicentric Castleman disease." Haematologica. 2023 Jan 1;108(1): 207-218. doi: 10.3324/haematol.2021.280370
2022
- Christian Hoffmann, et al. "Idiopathic Multicentric Castleman Disease Occurring Shortly after mRNA SARS-CoV-2 Vaccine." Vaccines (Basel). 2022 Oct 15;10(10):1725. doi: 10.3390/vaccines10101725
- David C. Fajgenbaum, et al. "The disease course of Castleman disease patients with fatal outcomes in the ACCELERATE registry." British Journal of Haematology. 2022 Jul; 198(2): 307-316. doi: 10.1111/bjh.18214
- Joshua D Brandstadter and David C Fajgenbaum. "How we manage idiopathic multicentric Castleman disease."
Clinical Advances in Hematology Oncology. 2022 Sep;20(9): 564-571.
- Sheila K. Pierson, et al. "CXCL13 is a predictive biomarker in idiopathic multicentric Castleman disease." Nature Communications. 2022 Nov 24; 13(1):7236. doi. 10.1038/s41467-022-34873-7
- Anna Wing, et al. "Transcriptome and unique cytokine microenvironment of Castleman disease." Modern Pathology. 2022 Oct 22;35(4): 451-461. doi: 10.1038/s41379-021-00950-3
- Elizaveta Belyaeva, et al. "Bone Marrow Findings of Idiopathic Multicentric Castleman Disease: A Histopathologic Analysis and Systematic Literature Review." Hematological Oncology. 2022 Feb 1;40(2): 191-201. doi: 10.1002/hon.2969
- Alexis D. Phillips, et al. "Increased mTORC2 pathway activation in lymph nodes of iMCD-TAFRO." Journal of Cellular & Molecular Medicine. 2022 Apr 30;26(11): 3174-3152. doi: 10.1111/jcmm.17251
- Mukherjee M, Martin R, et al. "Epidemiology and treatment patterns of idiopathic multicentric Castleman disease in the era of IL-6 directed therapy." Blood Adv. 2022 Jan 25;6(2): 359-367. doi: 10.1182/bloodadvances.2021004441
- Lu Zhang, et al. "A prospective, multicenter study of bortezomib, cyclophosphamide, and dexamethasone in relapsed/refractory iMCD." Leukemia & Lymphoma. 2022 Jan 16;63(3): 618-626. doi: 10.1080/10428194.2021.1999437
2021
- Goodman AM, Jeong AR, et al. "Novel somatic alterations in unicentric and idiopathic multicentric Castleman disease." European Journal of Hematology. 2021 Dec; 107(6): 642-649. doi: 10.1111/ejh.13702
- Nishimura Y, Fajgenbaum DC, et al. "Validated international definition of the thrombocytopenia, anasarca, fever, reticulin fibrosis, renal insufficiency, and organomegaly clinical subtype (TAFRO) of idiopathic multicentric Castleman disease." Am J Hematol. 2021 Oct 1;96(10): 1241-1252. doi: 10.1002/ajh.26292
- Pierson SK, Shenoy S, et al. "Discovery and validation of a novel subgroup and therapeutic target in idiopathic multicentric Castleman disease." Blood Adv. 2021 Sep 9;5(17): 3445-3456. doi: 10.1182/bloodadvances.2020004016.
- Lim JY, Duttke SH, et al. "DNMT3A haploinsufficiency causes dichotomous DNA methylation defects at enhancers in mature human immune cells." J Exp Med. 2021 May 10;218(7): e20202733. doi: 10.1084/jem.20202733
- Koa B, Borja AJ, et al. "Emerging role of 18F-FDG PET/CT in Castleman disease: a review." Insights Imaging. 2021 Mar 11;12(1): 35. doi: 10.1186/s13244-021-00963-1
- Zhang MY, Jia MN, et al. "UCD with MCD-like inflammatory state: surgical excision is highly effective." Blood Adv. 2021 Jan 12;5(1): 122-128. doi: 10.1182/bloodadvances.2020003607
- Kawano M, Hara S, et al. "HHV-8-negative multicentric Castleman disease patients with serological, histopathological and imaging features of IgG4-related disease." Rheumatology (Oxford). 2021 Jan 5;60(1): e3-e4. doi: 10.1093/rheumatology/keaa362
2020
- Abdallah H, Porterfield F, and Fajgenbaum DC. "Symptomatic relapse and long-term sequelae of COVID-19 in a previously healthy 30-year-old man." BMJ Case Reports. 2020 Dec 13;13(12): e239825. doi: 10.1136/bcr-2020-239825
- Fajgenbaum DC, Wu D, et al. Castleman Disease Collaborative Network Scientific Advisory Board diagnostic criteria international working group and treatment guidelines international working group. "Insufficient evidence exists to use histopathologic subtype to guide treatment of idiopathic multicentric Castleman disease." Am J Hematol. 2020 Dec;95(12): 1553-1561. doi: 10.1002/ajh.25992.
- Fajgenbaum DC and Phillips AD. "Commentary on A Case of Rapid Deterioration with Marked Hypergammaglobulinemia." Clin Chem. 2020 Nov 1;66(11): 1378-1379. doi: 10.1093/clinchem/hvaa156
- Fajgenbaum DC and Rader DJ. "Teaching Old Drugs New Tricks: Statins for COVID-19?" Cell Metab. 2020 Aug 4;32(2): 145-147. doi: 10.1016/j.cmet.2020.07.006.
- Fajgenbaum DC, Khor JS, et al. "Treatments Administered to the First 9152 Reported Cases of COVID-19: A Systematic Review." Infect Dis Ther. 2020 Sep;9(3): 435-449. doi: 10.1007/s40121-020-00303-8
- Pai RL, Japp AS, et al. "Type I IFN response associated with mTOR activation in the TAFRO subtype of idiopathic multicentric Castleman disease." JCI Insight. 2020 May 7;5(9): e135031. doi: 10.1172/jci.insight.135031
- Arenas DJ, Floess K, et al. "Increased mTOR activation in idiopathic multicentric Castleman disease." Blood. 2020 May 7;135(19): 1673-1684. doi: 10.1182/blood.2019002792
- Dispenzieri A and Fajgenbaum DC. "Overview of Castleman disease." Blood. 2020 Apr 16;135(16): 1353‐1364. doi: 10.1182/blood.2019000931
- Yoshimi A, Trippett TM, et al. "Genetic basis for iMCD-TAFRO." Oncogene. 2020 Feb 12;39(15): 3218‐3225. doi: 10.1038/s41388-020-1204-9
- Bernabei L, Waxman A, et al. "AA amyloidosis associated with Castleman disease: A case report and review of the literature." Medicine (Baltimore). 2020 Feb;99(6): e18978. doi: 10.1097/MD.0000000000018978
2019
- Suarez A, Reilly C, and Fajgenbaum DC. "Quantitative analysis of a rare disease network’s international contact database and E-repository provides insights into biobanking in the electronic consent era." Orphanet J Rare Dis. 2019 Jul 11;14(1): 173. doi: 10.1186/s13023-019-1145-y
- Fajgenbaum DC, Langan RA, et al. "Identifying and targeting pathogenic PI3K/AKT/mTOR signaling in IL-6-blockade-refractory idiopathic multicentric Castleman disease." J Clin Invest. 2019 Aug 13;129(10): 4451-4463. doi: 10.1172/JCI126091
- Nabel CS, Sameroff S. "Virome capture sequencing does not identify active viral infection in unicentric and idiopathic multicentric Castleman disease." PloS one. 2019 Jun 26;14(6): e0218660. doi: 10.1371/journal.pone.0218660
- Kobrin DM, Pinto AL, et al. "Letter to the editor regarding 'Non-cirrhotic portal hypertension associated with multicentric Castleman's disease: a case report'." Acta Oncol. 2019;58(4): 515‐517. doi: 10.1080/0284186X.2019.1574980
- Zhang L, ZHao AL, et al. "Phase 2 study using oral thalidomide-cyclophosphamide-prednisone for idiopathic multicentric Castleman disease." Blood. 2019 Apr 18;133(16): 1720-1728. doi: 10.1182/blood-2018-11-884577
- Zuccato M, Shilling D, and Fajgenbaum DC. "The Collaborative Network Approach: a model for advancing patient-centric research for Castleman disease and other rare diseases." Emerg Top Life Sci. 2019 Mar 29;3(1): 97-105. doi: 10.1042/ETLS20180178
- Amélie L, Gnemmi V, et al. "Renal pathologic findings in TAFRO syndrome: Is there a continuum between thrombotic microangiopathy and membranoproliferative glomerulonephritis? A case report and literature review." Front Immunol. 2019 Jun 28;10: 1489. doi: 10.3389/fimmu.2019.01489
- Morra DE, Pierson SK, et al. "Predictors of response to anti‐IL 6 monoclonal antibody therapy (siltuximab) in idiopathic multicentric Castleman disease: secondary analyses of phase II clinical trial data." British J Haematol. 2019 Jan;184(2): 232-241. doi: 10.1111/bjh.15588
2018
- Fajgenbaum DC. "Novel insights and therapeutic approaches in idiopathic multicentric Castleman disease." Blood. 2018 Nov 29;132(22): 2323-2330. doi: 10.1182/blood-2018-05-848671
- Van Rhee F, Vorhees P, et al. "International, evidence-based consensus treatment guidelines for idiopathic multicentric Castleman disease." Blood. 2018 Nov 15;132(20): 2115-2124. doi: 10.1182/blood-2018-07-862334
- Dong Y, Zhang L, et al. "Effectiveness of rituximab-containing treatment regimens in idiopathic multicentric Castleman disease." Ann Hematol. 2018 Sep;97(9): 1641-1647. doi: 10.1007/s00277-018-3347-0
- Baker TS, Gambino K, et al. "A novel FAS mutation with variable expressivity in a family with unicentric and idiopathic multicentric Castleman disease." Blood Adv. 2018 Nov 13;2(21): 2959-2963. doi: 10.1182/bloodadvances.2018023911
- Pierson SK, Stonestrom AJ, et al. "Plasma proteomics identifies a 'chemokine storm' in idiopathic multicentric Castleman disease." Am J Hematol. 2018 Jul;93(7): 902-912. doi: 10.1002/ajh.25123
- Lee J, Werth VP, et al. "Perspective from the 5th international pemphigus and pemphigoid foundation scientific conference." Front Med (Lausanne). 2018 Nov 8;5: 306. doi: 10.3389/fmed.2018.00306
- Fajgenbaum DC, Shilling D. "Castleman Disease Pathogenesis." Hematol Oncol Clin North Am. 2018 Feb;32(1): 11-21. doi: 10.1016/j.hoc.2017.09.002
- Simon S, Fajgenbaum DC, et al. "Schnitzler syndrome co-occurring with idiopathic multicentric Castleman disease that responds to anti-IL-1 therapy: A case report and clue to pathophysiology." Curr Res Transl Med. 2018 Sep;66(3): 83-86. doi: 10.1016/j.retram.2018.06.001
2017
- Louis C, Vijgen S, et al. "TAFRO Syndrome in Caucasians: A Case Report and Review of the Literature." Front Med (Lausanne). 2017 Sep 22;4: 149. doi: 10.3389/fmed.2017.00149
- Srkalovic G, Marijanovic I, et al. "TAFRO syndrome: New subtype of idiopathic multicentric Castleman disease." Bosn J Basic Med Sci. 2017 May 20;17(2): 81-84. doi: 10.17305/bjbms.2017.1930
- Yu L, Tu M, et al. "Clinical and pathological characteristics of HIV- and HHV-8-negative Castleman disease." Blood. 2017 Mar 23;129(12): 1658-1668. doi: 10.1182/blood-2016-11-748855
- Fajgenbaum DC, Uldrick TS, et al."International, evidence-based consensus diagnostic criteria for HHV-8-negative/idiopathic multicentric Castleman disease." 2017 Mar 23;129(12): 1646-1657. doi: 10.1182/blood-2016-10-746933
- Behnia F, Elojeimy S, et al. "Potential value of FDG PET-CT in diagnosis and follow-up of TAFRO syndrome." Ann Hematol. 2017 Mar;96(3): 497-500. doi: 10.1007/s00277-016-2875-8
2016
- Liu AY, Nabel CS, et al. "Idiopathic multicentric Castleman's disease: a systematic literature review." 2016 Apr;3(4): e163-75. doi: 10.1016/S2352-3026(16)00006-5
- Fajgenbaum DC, Ruth JR, et al. "The collaborative network approach: a new framework to accelerate Castleman's disease and other rare disease research." 2016 Apr;3(4): e150-2. doi: 10.1016/S2352-3026(16)00007-7
- Iwaki N, Fajgenbaum DC, et al. "Clinicopathologic analysis of TAFRO syndrome demonstrates a distinct subtype of HHV-8-negative multicentric Castleman disease." Am J Hematol. 2016 Feb;91(2): 220-6. doi: 10.1002/ajh.24242
- Fajgenbaum DC and Kurzrock R. "Siltuximab: a targeted therapy for idiopathic multicentric Castleman disease." Immunotherapy. 2016;8(1): 17-26. doi: 10.2217/imt.15.95
2015
- Newman SK, Jayanthan RK, et al. "Taking Control of Castleman Disease: Leveraging Precision Medicine Technologies to Accelerate Rare Disease Research." Yale J Biol Med. 2015;88(4): 383‐388.
2014
- Fajgenbaum DC, van Rhee F, and Nabel CS. "HHV-8-negative, idiopathic multicentric Castleman disease: novel insights into biology, pathogenesis, and therapy." Blood. 2014 May 8;123(19): 2924-33. doi: 10.1182/blood-2013-12-545087
- Fajgenbaum DC, Rosenbach M, et al. "Eruptive cherry hemangiomatosis associated with multicentric Castleman disease: a case report and diagnostic clue" [published correction appears in JAMA Dermatol. 2014 Apr;150(4): 460. Fajgenbaum David, [corrected to Fajgenbaum, David C]]. JAMA Dermatol. 2013;149(2):204‐208.