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Idiopathic Multicentric Castleman Disease (iMCD) — References

Curated PubMed references organized by topic for the iMCD QSP model. All references are HHV-8-negative (idiopathic) MCD unless otherwise noted.

1. Diagnostic Criteria & Clinical Subtypes

  1. Fajgenbaum DC, Uldrick TS, Bagg A, et al. International, evidence-based consensus diagnostic criteria for HHV-8-negative/idiopathic multicentric Castleman disease. Blood. 2017;129(12):1646-1657. https://pubmed.ncbi.nlm.nih.gov/28087540/
  2. Iwaki N, Fajgenbaum DC, Nabel CS, et al. Clinicopathologic analysis of TAFRO syndrome demonstrates a distinct subtype of HHV-8-negative multicentric Castleman disease. Am J Hematol. 2016;91(2):220-226. https://pubmed.ncbi.nlm.nih.gov/26805758/
  3. Dispenzieri A, Fajgenbaum DC. Overview of Castleman disease. Blood. 2020;135(16):1353-1364. https://pubmed.ncbi.nlm.nih.gov/32106302/
  4. Liu AY, Nabel CS, Finkelman BS, et al. Idiopathic multicentric Castleman's disease: a systematic literature review. Lancet Haematol. 2016;3(4):e163-e175. https://pubmed.ncbi.nlm.nih.gov/27063975/
  5. Masaki Y, Kawabata H, Takai K, et al. Proposed diagnostic criteria, disease severity classification and treatment strategy for TAFRO syndrome, 2015 version. Int J Hematol. 2016;103(6):686-692. https://pubmed.ncbi.nlm.nih.gov/27084250/

2. Pathophysiology — IL-6 Axis & Cytokine Storm

  1. Yoshizaki K, Matsuda T, Nishimoto N, et al. Pathogenic significance of interleukin-6 (IL-6/BSF-2) in Castleman's disease. Blood. 1989;74(4):1360-1367. https://pubmed.ncbi.nlm.nih.gov/2788466/
  2. Beck JT, Hsu SM, Wijdenes J, et al. Brief report: alleviation of systemic manifestations of Castleman's disease by monoclonal anti-interleukin-6 antibody. N Engl J Med. 1994;330(9):602-605. https://pubmed.ncbi.nlm.nih.gov/8302342/
  3. Kishimoto T, Akira S, Narazaki M, Taga T. Interleukin-6 family of cytokines and gp130. Blood. 1995;86(4):1243-1254. https://pubmed.ncbi.nlm.nih.gov/7632928/
  4. Garbers C, Heink S, Korn T, Rose-John S. Interleukin-6: designing specific therapeutics for a complex cytokine. Nat Rev Drug Discov. 2018;17(6):395-412. https://pubmed.ncbi.nlm.nih.gov/29725131/
  5. Hunter CA, Jones SA. IL-6 as a keystone cytokine in health and disease. Nat Immunol. 2015;16(5):448-457. https://pubmed.ncbi.nlm.nih.gov/25898198/
  6. Fajgenbaum DC, June CH. Cytokine storm. N Engl J Med. 2020;383(23):2255-2273. https://pubmed.ncbi.nlm.nih.gov/33264547/

3. Pathophysiology — Beyond IL-6 (mTOR / VEGF / NLRP3)

  1. Fajgenbaum DC, Langan RA, Japp AS, et al. Identifying and targeting pathogenic PI3K/AKT/mTOR signaling in IL-6-blockade-refractory iMCD. J Clin Invest. 2019;129(10):4451-4463. https://pubmed.ncbi.nlm.nih.gov/31408438/
  2. Pierson SK, Khor JS, Ziglar J, et al. ACCELERATE: a patient-powered natural history study design enabling clinical and therapeutic discoveries in a rare disorder. Cell Rep Med. 2020;1(9):100158. https://pubmed.ncbi.nlm.nih.gov/33377128/
  3. Nishi J, Maruyama I. Increased expression of vascular endothelial growth factor (VEGF) in Castleman's disease. Pathol Int. 2000;50(3):187-191. https://pubmed.ncbi.nlm.nih.gov/10792783/
  4. Pierson SK, Stonestrom AJ, Shilling D, et al. Plasma proteomics identifies a 'chemokine storm' in idiopathic multicentric Castleman disease. Am J Hematol. 2018;93(7):902-912. https://pubmed.ncbi.nlm.nih.gov/29675946/
  5. Wing A, Xu J, Meng W, et al. Transcriptome and unique cytokine microenvironment of Castleman disease. Mod Pathol. 2022;35(4):451-461. https://pubmed.ncbi.nlm.nih.gov/34732847/

4. Lymph Node Histopathology

  1. Frizzera G, Peterson BA, Bayrd ED, Goldman A. A systemic lymphoproliferative disorder with morphologic features of Castleman's disease: clinical findings and clinicopathologic correlations in 15 patients. J Clin Oncol. 1985;3(9):1202-1216. https://pubmed.ncbi.nlm.nih.gov/4031968/
  2. Lee FC, Merchant SH. Alleviation of systemic manifestations of multicentric Castleman's disease by thalidomide. Am J Hematol. 2003;73(1):48-53. https://pubmed.ncbi.nlm.nih.gov/12701121/
  3. Wang HW, Pittaluga S, Jaffe ES. Multicentric Castleman disease: where are we now? Semin Diagn Pathol. 2016;33(5):294-306. https://pubmed.ncbi.nlm.nih.gov/27296355/
  4. Cronin DM, Warnke RA. Castleman disease: an update on classification and the spectrum of associated lesions. Adv Anat Pathol. 2009;16(4):236-246. https://pubmed.ncbi.nlm.nih.gov/19546611/

5. Treatment — Siltuximab (Approved)

  1. van Rhee F, Wong RS, Munshi N, et al. Siltuximab for multicentric Castleman's disease: a randomised, double-blind, placebo-controlled trial. Lancet Oncol. 2014;15(9):966-974. https://pubmed.ncbi.nlm.nih.gov/25042199/ (CONCERT trial)
  2. Kurzrock R, Voorhees PM, Casper C, et al. A phase I, open-label study of siltuximab, an anti-IL-6 monoclonal antibody, in patients with B-cell non-Hodgkin lymphoma, multiple myeloma, or Castleman disease. Clin Cancer Res. 2013;19(13):3659-3670. https://pubmed.ncbi.nlm.nih.gov/23659971/
  3. van Rhee F, Casper C, Voorhees PM, et al. A phase 2, open-label, multicenter study of the long-term safety of siltuximab (anti-IL-6 monoclonal antibody) in patients with multicentric Castleman disease. Oncotarget. 2015;6(30):30408-30419. https://pubmed.ncbi.nlm.nih.gov/26327301/
  4. Markham A, Patel T. Siltuximab: first global approval. Drugs. 2014;74(10):1175-1179. https://pubmed.ncbi.nlm.nih.gov/25022950/
  5. van Rhee F, Voorhees P, Dispenzieri A, et al. International, evidence-based consensus treatment guidelines for idiopathic multicentric Castleman disease. Blood. 2018;132(20):2115-2124. https://pubmed.ncbi.nlm.nih.gov/30181172/

6. Treatment — Tocilizumab

  1. Nishimoto N, Kanakura Y, Aozasa K, et al. Humanized anti-interleukin-6 receptor antibody treatment of multicentric Castleman disease. Blood. 2005;106(8):2627-2632. https://pubmed.ncbi.nlm.nih.gov/15998837/
  2. Nishimoto N, Sasai M, Shima Y, et al. Improvement in Castleman's disease by humanized anti-interleukin-6 receptor antibody therapy. Blood. 2000;95(1):56-61. https://pubmed.ncbi.nlm.nih.gov/10607684/
  3. Frey N, Grange S, Woodworth T. Population pharmacokinetic analysis of tocilizumab in patients with rheumatoid arthritis. J Clin Pharmacol. 2010;50(7):754-766. https://pubmed.ncbi.nlm.nih.gov/20097933/
  4. Matsuyama M, Suzuki T, Tsuboi H, et al. Anti-interleukin-6 receptor antibody (tocilizumab) treatment of multicentric Castleman's disease. Intern Med. 2007;46(11):771-774. https://pubmed.ncbi.nlm.nih.gov/17541233/

7. Treatment — Sirolimus / mTOR (TAFRO subtype)

  1. Fajgenbaum DC, Langan RA, Japp AS, et al. Identifying and targeting pathogenic PI3K/AKT/mTOR signaling in IL-6-blockade-refractory iMCD. J Clin Invest. 2019;129(10):4451-4463. https://pubmed.ncbi.nlm.nih.gov/31408438/
  2. Arenas DJ, Floess K, Kobrin D, et al. Increased mTOR activation in idiopathic multicentric Castleman disease. Blood. 2020;135(19):1673-1684. https://pubmed.ncbi.nlm.nih.gov/32206779/
  3. MacDonald A, Scarola J, Burke JT, Zimmerman JJ. Clinical pharmacokinetics and therapeutic drug monitoring of sirolimus. Clin Ther. 2000;22 Suppl B:B101-B121. https://pubmed.ncbi.nlm.nih.gov/10823376/

8. Treatment — Rituximab / Anakinra / Other

  1. Ide M, Kawachi Y, Izumi Y, et al. Long-term remission in HIV-negative patients with multicentric Castleman's disease using rituximab. Eur J Haematol. 2006;76(2):119-123. https://pubmed.ncbi.nlm.nih.gov/16405435/
  2. El-Osta HE, Kurzrock R. Castleman's disease: from basic mechanisms to molecular therapeutics. Oncologist. 2011;16(4):497-511. https://pubmed.ncbi.nlm.nih.gov/21441295/
  3. Galeotti C, Tran TA, Franchi-Abella S, Fabre M, Pariente D, Koné-Paut I. IL-1 receptor antagonist (anakinra) as a treatment of corticodependent Castleman's disease. Pediatrics. 2008;121(2):e441-e444. https://pubmed.ncbi.nlm.nih.gov/18193992/
  4. Pierson SK, Shenoy S, Oromendia AB, et al. Discovery and validation of a novel subgroup and therapeutic target in idiopathic multicentric Castleman disease. Blood Adv. 2021;5(17):3445-3456. https://pubmed.ncbi.nlm.nih.gov/34438448/

9. Treatment — CHOP / Chemotherapy / Salvage

  1. Bower M, Newsom-Davis T, Naresh K, et al. Clinical features and outcome in HIV-associated multicentric Castleman's disease. J Clin Oncol. 2011;29(18):2481-2486. https://pubmed.ncbi.nlm.nih.gov/21555697/
  2. Talat N, Belgaumkar AP, Schulte KM. Surgery in Castleman's disease: a systematic review of 404 published cases. Ann Surg. 2012;255(4):677-684. https://pubmed.ncbi.nlm.nih.gov/22367441/
  3. Pierson SK, Lim MS, Srkalovic G, et al. Treatment consistency with consensus guidelines is associated with improved outcomes in iMCD. Blood Adv. 2023;7(21):6652-6664. https://pubmed.ncbi.nlm.nih.gov/37466574/

10. Calibration & Population PK

  1. Puchalski T, Prabhakar U, Jiao Q, Berns B, Davis HM. Pharmacokinetic and pharmacodynamic modeling of an anti-interleukin-6 chimeric monoclonal antibody (siltuximab) in patients with metastatic renal cell carcinoma. Clin Cancer Res. 2010;16(5):1652-1661. https://pubmed.ncbi.nlm.nih.gov/20179212/
  2. Saito Y, Maruyama T, Ozasa H, et al. Population pharmacokinetic analysis of tocilizumab in adult and paediatric patients. Br J Clin Pharmacol. 2017;83(5):968-979. https://pubmed.ncbi.nlm.nih.gov/27800645/
  3. Sumimoto S, Kawai M, Kasajima Y, Hamamoto T. Aggressive form of Castleman's disease: discrepancy between clinical improvement and pathological findings. Acta Paediatr Jpn. 1990;32(4):396-400. https://pubmed.ncbi.nlm.nih.gov/2123581/

11. Outcomes, Survival & Long-term Follow-up

  1. Powles T, Stebbing J, Bazeos A, et al. The role of immune suppression and HHV-8 in the increasing incidence of HIV-associated multicentric Castleman's disease. Ann Oncol. 2009;20(4):775-779. https://pubmed.ncbi.nlm.nih.gov/19179554/
  2. Mukherjee S, Martin R, Sande B, et al. Epidemiology and treatment patterns of idiopathic multicentric Castleman disease in the era of IL-6-directed therapy. Blood Adv. 2022;6(2):359-367. https://pubmed.ncbi.nlm.nih.gov/34638137/
  3. Fajgenbaum DC, Wu D, Goodman A, et al. Insufficient evidence exists to use histopathologic subtype to guide treatment of idiopathic multicentric Castleman disease. Am J Hematol. 2020;95(12):1553-1561. https://pubmed.ncbi.nlm.nih.gov/32894785/

12. Reviews & Recent Advances

  1. Fajgenbaum DC. Novel insights and therapeutic approaches in idiopathic multicentric Castleman disease. Blood. 2018;132(22):2323-2330. https://pubmed.ncbi.nlm.nih.gov/30487129/
  2. Carbone A, Borok M, Damania B, et al. Castleman disease. Nat Rev Dis Primers. 2021;7(1):84. https://pubmed.ncbi.nlm.nih.gov/34824298/
  3. Soumerai JD, Sohani AR, Abramson JS. Diagnosis and management of Castleman disease. Cancer Control. 2014;21(4):266-278. https://pubmed.ncbi.nlm.nih.gov/25310208/
  4. Yoshizaki K, Murayama S, Ito H, Koga T. The role of interleukin-6 in Castleman disease. Hematol Oncol Clin North Am. 2018;32(1):23-36. https://pubmed.ncbi.nlm.nih.gov/29157616/
  5. Munshi N, Mehra M, van de Velde H, Desai A, Potluri R, Vermeulen J. Use of a claims database to characterize and estimate the incidence rate for Castleman disease. Leuk Lymphoma. 2015;56(5):1252-1260. https://pubmed.ncbi.nlm.nih.gov/25120049/

13. Related QSP / Cytokine Modeling References

  1. Hunter CA, Jones SA. IL-6 as a keystone cytokine in health and disease. Nat Immunol. 2015;16(5):448-457. https://pubmed.ncbi.nlm.nih.gov/25898198/
  2. Calabrese LH, Rose-John S. IL-6 biology: implications for clinical targeting in rheumatic disease. Nat Rev Rheumatol. 2014;10(12):720-727. https://pubmed.ncbi.nlm.nih.gov/25136784/
  3. Mihara M, Hashizume M, Yoshida H, Suzuki M, Shiina M. IL-6/IL-6 receptor system and its role in physiological and pathological conditions. Clin Sci (Lond). 2012;122(4):143-159. https://pubmed.ncbi.nlm.nih.gov/22029668/

14. Hepcidin / Anemia of Inflammation

  1. Ganz T. Hepcidin and iron regulation, 10 years later. Blood. 2011;117(17):4425-4433. https://pubmed.ncbi.nlm.nih.gov/21346250/
  2. Weiss G, Ganz T, Goodnough LT. Anemia of inflammation. Blood. 2019;133(1):40-50. https://pubmed.ncbi.nlm.nih.gov/30401705/

15. Diagnostic Biomarkers

  1. Hoffmann C, Hentrich M, Tiemann M, et al. Long-term outcome of patients with HIV-associated multicentric Castleman disease. Blood. 2011;118(13):3499-3503. https://pubmed.ncbi.nlm.nih.gov/21778341/
  2. Stone K, Woods E, Szmania SM, et al. Interleukin-6 receptor polymorphism is prevalent in HIV-negative Castleman disease and is associated with increased soluble interleukin-6 receptor levels. PLoS One. 2013;8(1):e54610. https://pubmed.ncbi.nlm.nih.gov/23372742/
  3. Mukherjee S, Kanhai K, Kauffman D, et al. Epidemiology of idiopathic multicentric Castleman disease in the era of IL-6 targeted therapy. Blood Adv. 2022;6(2):359-367. https://pubmed.ncbi.nlm.nih.gov/34638137/

16. Patient-Reported Outcomes & Symptoms

  1. Casper C, Chaturvedi S, Munshi N, et al. Analysis of inflammatory and anemia-related biomarkers in a randomized, double-blind, placebo-controlled study of siltuximab. Clin Cancer Res. 2015;21(19):4294-4304. https://pubmed.ncbi.nlm.nih.gov/26124203/
  2. Pierson SK, Khor JS, Ziglar J, et al. Symptom burden, treatment patterns, and management of patients with idiopathic multicentric Castleman disease in ACCELERATE. Br J Haematol. 2023;201(6):e9-e12. https://pubmed.ncbi.nlm.nih.gov/36864759/

Note on Model Calibration

The mrgsolve parameters in imcd_mrgsolve_model.R were anchored to:

  • Siltuximab PopPK — Puchalski 2010 Clin Cancer Res, van Rhee 2014 Lancet Oncol (CONCERT)
  • Tocilizumab PopPK — Frey 2010 J Clin Pharmacol, Saito 2017 Br J Clin Pharmacol
  • Sirolimus PopPK — MacDonald 2000 Clin Ther
  • CRP/IL-6 dynamics — Casper 2015 Clin Cancer Res biomarker substudy
  • TAFRO mTOR rationale — Fajgenbaum 2019 JCI, Arenas 2020 Blood
  • Treatment guideline anchors — van Rhee 2018 Blood (consensus), Pierson 2023 Blood Adv (real-world)

CDCN = Castleman Disease Collaborative Network · ACCELERATE = patient-powered natural history registry.