POEMS syndrome medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief: Sabawoon Mirwais, M.B.B.S, M.D.[2]

Overview

Medical Therapy

  • Patients with isolated bone lesion without bone marrow clonal plasma cells involvement can be treated with radiotherapy.[1]
  • Patients with a disseminated disease (more bone lesions and/or bone marrow plasmacytosis) are the candidates for systemic therapy.[1]

Radiotherapy

  • More than 50% of patients treated with radiotherapy show a significant improvement of neuropathy.[2]
  • Maximal response to radiotherapy, i.e., improvement of neuropathy, can not be attained until 2–3 years since the first evidence of the effect of therapy.[3][4]
  • Improvement of anasarca, papilledema, pulmonary hypertension and skin changes may occur within months after the end of radiotherapy.[3][4]
  • Radiotherapy can be considered as primary therapy if the bone lesion is large despite an infiltrated bone marrow.

Systemic Therapy

Corticosteroids

  • Corticosteroids can attenuate the symptoms for a short time without affecting the progression of the syndrome.[5]

Autologous Stem Cell Transplantation (ASCT)

  • It is considered the preferred initial therapy in young patients.[6]
  • It is associated with a durable response even if patients experience a relapse of the disease.[6]
  • A retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation showed the 1-year non-relapse mortality to be 3.3%.[7]
  • Young patients can be ineligible for ASCT in the presence of serous cavity effusion, renal impairment, pulmonary hypertension, and severe end-organ dysfunction.
  • It is a lethal procedure and can result in engraftment syndrome (fever, rash, diarrhea, weight gain, and respiratory problems that occur 7–15 days since stem cell infusion).[8]

Induction Therapy

  • Induction therapy involves cyclophosphamide or lenalidomide, thalidomide or bortezomib in combination with high-dose dexamethasone.[9]
  • In patients with an overproduction of IL-12 and VEGF, induction therapy can modify the hypercytokinemia status, improving clinical condition and can control disease severity making patients eligible for ASCT.[9]
  • The use of induction therapy before ASCT can also decrease the incidence of peritransplant complications.[10][11]
  • Induction therapy before mobilization (i.e., stimulation of stem cells out of the bone marrow into the bloodstream) reduces the levels of cytokines and also reduces the risk of inadequate mobilization.

Alkylator-base Therapy

  • It is the treatment of choice for patients not suitable for ASCT.
  • It consists of melphalan or cyclophosphamide plus corticosteroids.[2]
  • It is associated with a 2-year overall survival (OS) rate of 78%.[2]
  • Adverse effects of this therapy include hematologic neoplasia such as leukemia and myelodysplasia.
  • In the first prospective clinical trial, 81% of the patients showed hematologic response and 100% of the patients showed improvement in neurological symptoms and reduction on VEGF levels.[12]
  • Data on the long term effect of this therapy is still unavailable.[12]

Thalidomide

  • It has been tested in POEMS syndrome due to its antiangiogenic, anti-inflammatory, and immunomodulating properties.
  • A multicenter, randomized, double-blind study showed a reduction of VEGF serum level with thalidomide.[13][14]
  • Neurotoxicity, a serious side effect, limits the use of thalidomide.[15][14]

Lenalidomide

  • It appears to be the most promising drug for the treatment of POEMS syndrome.
  • It has been reported to have a long-lasting response and good tolerability.[16][17][18][19][20]
  • A retrospective pooled analysis of 51 subjects treated with lenalidomide showed a 12-month progression free survival (PFS) rate of 93% and a 24-month rate of 47%.[21]
  • Lenalidomide, as an induction therapy before the transplant, can improve the patient clinical status and decrease transplant-related morbidity.

Bortezomib

  • Bortezomib, alone or in combination, can be used in newly-diagnosed and in relapsed patients.[22][23][24]
  • It is associated with highly satisfactory responses on neuropathy, serum VEGF level, and extravascular overload.[22][23][25][24]
  • It is, however, also associated with a potential risk of progression of existing neuropathy.

Bevacizumab

  • Bevacizumab has anti-VEGF properties and can result in a rapid decrease in the serum VEGF levels.[26]
  • It is not, however, associated with clinical improvement.[26]



References

  1. ↑ 1.0 1.1 Dispenzieri A (June 2012). "How I treat POEMS syndrome". Blood. 119 (24): 5650–8. doi:10.1182/blood-2012-03-378992. PMC 3425020. PMID 22547581.
  2. ↑ 2.0 2.1 2.2 Dispenzieri A, Kyle RA, Lacy MQ, Rajkumar SV, Therneau TM, Larson DR, Greipp PR, Witzig TE, Basu R, Suarez GA, Fonseca R, Lust JA, Gertz MA (April 2003). "POEMS syndrome: definitions and long-term outcome". Blood. 101 (7): 2496–506. doi:10.1182/blood-2002-07-2299. PMID 12456500.
  3. ↑ 3.0 3.1 Brewis MJ, Church AC, Peacock AJ, Thomson S, Tighe J, Johnson MK (December 2014). "Pulmonary hypertension in POEMS syndrome: resolution following radiotherapy". Pulm Circ. 4 (4): 732–5. doi:10.1086/678553. PMC 4278633. PMID 25610609.
  4. ↑ 4.0 4.1 Dispenzieri A (February 2014). "POEMS syndrome: 2014 update on diagnosis, risk-stratification, and management". Am. J. Hematol. 89 (2): 214–23. doi:10.1002/ajh.23644. PMID 24532337.
  5. ↑ Dispenzieri A (October 2015). "POEMS syndrome: update on diagnosis, risk-stratification, and management". Am. J. Hematol. 90 (10): 951–62. doi:10.1002/ajh.24171. PMID 26331353.
  6. ↑ 6.0 6.1 D'Souza A, Lacy M, Gertz M, Kumar S, Buadi F, Hayman S, Dingli D, Zeldenrust S, Kyle R, Ansell S, Inwards D, Johnston P, Micallef I, Porrata L, Litzow M, Gastineau D, Hogan W, Dispenzieri A (July 2012). "Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): a single-center experience". Blood. 120 (1): 56–62. doi:10.1182/blood-2012-04-423178. PMID 22611150.
  7. ↑ Cook G, Iacobelli S, van Biezen A, Ziagkos D, LeBlond V, Abraham J, McQuaker G, Schoenland S, Rambaldi A, Halaburda K, Rovira M, Sica S, Byrne J, Sanz RG, Nagler A, van de Donk NW, Sinisalo M, Cook M, Kröger N, De Witte T, Morris C, Garderet L (January 2017). "High-dose therapy and autologous stem cell transplantation in patients with POEMS syndrome: a retrospective study of the Plasma Cell Disorder sub-committee of the Chronic Malignancy Working Party of the European Society for Blood & Marrow Transplantation". Haematologica. 102 (1): 160–167. doi:10.3324/haematol.2016.148460. PMC 5210246. PMID 27634201.
  8. ↑ Dispenzieri A, Lacy MQ, Hayman SR, Kumar SK, Buadi F, Dingli D, Litzow MR, Gastineau DA, Inwards DJ, Elliott MA, Micallef IN, Ansell SM, Hogan WJ, Porrata LF, Johnston PA, Afessa B, Bryce A, Kyle RA, Gertz MA (May 2008). "Peripheral blood stem cell transplant for POEMS syndrome is associated with high rates of engraftment syndrome". Eur. J. Haematol. 80 (5): 397–406. doi:10.1111/j.1600-0609.2008.01037.x. PMC 2327207. PMID 18221391.
  9. ↑ 9.0 9.1 Kanai K, Sawai S, Sogawa K, Mori M, Misawa S, Shibuya K, Isose S, Fujimaki Y, Noto Y, Sekiguchi Y, Nasu S, Nakaseko C, Takano S, Yoshitomi H, Miyazaki M, Nomura F, Kuwabara S (August 2012). "Markedly upregulated serum interleukin-12 as a novel biomarker in POEMS syndrome". Neurology. 79 (6): 575–82. doi:10.1212/WNL.0b013e318263c42b. PMID 22843279.
  10. ↑ Jimenez-Zepeda VH, Trudel S, Reece DE, Chen C, Rabea AM, Kukreti V (October 2011). "Cyclophosphamide and prednisone induction followed by cyclophosphamide mobilization effectively decreases the incidence of engraftment syndrome in patients with POEMS syndrome who undergo stem cell transplantation". Am. J. Hematol. 86 (10): 873–5. doi:10.1002/ajh.22115. PMID 21815185.
  11. ↑ Li J, Zhang W, Duan MH, Jiao L, Zhu TN, Han B, Zhang L, Gan J, Zhou DB (February 2013). "PBSC mobilization in newly diagnosed patients with POEMS syndrome: outcomes and prognostic factors". Bone Marrow Transplant. 48 (2): 233–7. doi:10.1038/bmt.2012.138. PMID 22825426.
  12. ↑ 12.0 12.1 Li J, Zhang W, Jiao L, Duan MH, Guan HZ, Zhu WG, Tian Z, Zhou DB (June 2011). "Combination of melphalan and dexamethasone for patients with newly diagnosed POEMS syndrome". Blood. 117 (24): 6445–9. doi:10.1182/blood-2010-12-328112. PMC 3123016. PMID 21393478.
  13. ↑ Misawa S, Sato Y, Katayama K, Nagashima K, Aoyagi R, Sekiguchi Y, Sobue G, Koike H, Yabe I, Sasaki H, Watanabe O, Takashima H, Nishizawa M, Kawachi I, Kusunoki S, Mitsui Y, Kikuchi S, Nakashima I, Ikeda S, Kohara N, Kanda T, Kira J, Hanaoka H, Kuwabara S (October 2016). "Safety and efficacy of thalidomide in patients with POEMS syndrome: a multicentre, randomised, double-blind, placebo-controlled trial". Lancet Neurol. 15 (11): 1129–37. doi:10.1016/S1474-4422(16)30157-0. PMID 27496680.
  14. ↑ 14.0 14.1 Kuwabara S, Misawa S, Kanai K, Sawai S, Hattori T, Nishimura M, Nakaseko C (November 2008). "Thalidomide reduces serum VEGF levels and improves peripheral neuropathy in POEMS syndrome". J. Neurol. Neurosurg. Psychiatry. 79 (11): 1255–7. doi:10.1136/jnnp.2008.150177. PMID 18469028.
  15. ↑ Sinisalo M, Hietaharju A, Sauranen J, Wirta O (May 2004). "Thalidomide in POEMS syndrome: case report". Am. J. Hematol. 76 (1): 66–8. doi:10.1002/ajh.20051. PMID 15114600.
  16. ↑ Dispenzieri A, Klein CJ, Mauermann ML (August 2007). "Lenalidomide therapy in a patient with POEMS syndrome". Blood. 110 (3): 1075–6. doi:10.1182/blood-2007-03-082354. PMID 17644745.
  17. ↑ Royer B, Merlusca L, Abraham J, Musset L, Haroche J, Choquet S, Leleu X, Sebban C, Decaux O, Galicier L, Roussel M, Recher C, Banos A, Guichard I, Brisseau JM, Godmer P, Hermine O, Deplanque G, Facon T, Asli B, Leblond V, Fermand JP, Marolleau JP, Jaccard A (March 2013). "Efficacy of lenalidomide in POEMS syndrome: a retrospective study of 20 patients". Am. J. Hematol. 88 (3): 207–12. doi:10.1002/ajh.23374. PMID 23335406.
  18. ↑ Vannata B, Laurenti L, Chiusolo P, Sorà F, Balducci M, Sabatelli M, Luigetti M, Giannotta C, De Stefano V, Leone G, Sica S (June 2012). "Efficacy of lenalidomide plus dexamethasone for POEMS syndrome relapsed after autologous peripheral stem-cell transplantation". Am. J. Hematol. 87 (6): 641–2. doi:10.1002/ajh.23195. PMID 22488443.
  19. ↑ Suyanı E, Yağcı M, Sucak GT (2011). "Complete remission with a combination of lenalidomide, cyclophosphamide and prednisolone in a patient with incomplete POEMS syndrome". Acta Haematol. 126 (4): 199–201. doi:10.1159/000329896. PMID 21849771.
  20. ↑ Cai QQ, Wang C, Cao XX, Cai H, Zhou DB, Li J (October 2015). "Efficacy and safety of low-dose lenalidomide plus dexamethasone in patients with relapsed or refractory POEMS syndrome". Eur. J. Haematol. 95 (4): 325–30. doi:10.1111/ejh.12492. PMID 25401269.
  21. ↑ Zagouri F, Kastritis E, Gavriatopoulou M, Sergentanis TN, Psaltopoulou T, Terpos E, Dimopoulos MA (September 2014). "Lenalidomide in patients with POEMS syndrome: a systematic review and pooled analysis". Leuk. Lymphoma. 55 (9): 2018–23. doi:10.3109/10428194.2013.869329. PMID 24295131.
  22. ↑ 22.0 22.1 Tang X, Shi X, Sun A, Qiu H, Gu B, Zhou H, Xue S, Liu Y, Ruan C, Wu D (December 2009). "Successful bortezomib-based treatment in POEMS syndrome". Eur. J. Haematol. 83 (6): 609–10. doi:10.1111/j.1600-0609.2009.01330.x. PMID 19674063.
  23. ↑ 23.0 23.1 Kaygusuz I, Tezcan H, Cetiner M, Kocakaya O, Uzay A, Bayik M (February 2010). "Bortezomib: a new therapeutic option for POEMS syndrome". Eur. J. Haematol. 84 (2): 175–7. doi:10.1111/j.1600-0609.2009.01341.x. PMID 19732138.
  24. ↑ 24.0 24.1 Warsame R, Kohut IE, Dispenzieri A (June 2012). "Successful use of cyclophosphamide, bortezomib, and dexamethasone to treat a case of relapsed POEMS". Eur. J. Haematol. 88 (6): 549–50. doi:10.1111/j.1600-0609.2012.01780.x. PMID 22416898.
  25. ↑ Ohguchi H, Ohba R, Onishi Y, Fukuhara N, Okitsu Y, Yamamoto J, Ishizawa K, Ichinohasama R, Harigae H (September 2011). "Successful treatment with bortezomib and thalidomide for POEMS syndrome". Ann. Hematol. 90 (9): 1113–4. doi:10.1007/s00277-010-1133-8. PMID 21153416.
  26. ↑ 26.0 26.1 Sekiguchi Y, Misawa S, Shibuya K, Nasu S, Mitsuma S, Iwai Y, Beppu M, Sawai S, Ito S, Hirano S, Nakaseko C, Kuwabara S (December 2013). "Ambiguous effects of anti-VEGF monoclonal antibody (bevacizumab) for POEMS syndrome". J. Neurol. Neurosurg. Psychiatry. 84 (12): 1346–8. doi:10.1136/jnnp-2012-304874. PMID 23463868.

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