Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance

Current Opinion in Neurology

Volumn 30, Issue 5, 2017, Pages 457-463

  Uruha, Akinori ^a   Benveniste, Olivier ^b  

^a SORBONNE UNIVERSITÉ   (France)

^b ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS   (France)

Author keywords

autologous stem cell transplantation following high dose melphalan; head drop; monoclonal gammopathy of undetermined significance; nemaline body

Indexed keywords

MELPHALAN;

AUTOLOGOUS STEM CELL TRANSPLANTATION; CLINICAL FEATURE; DISEASE ASSOCIATION; DYSARTHRIA; DYSPHAGIA; HUMAN; LATE ONSET DISORDER; MONOCLONAL IMMUNOGLOBULINEMIA; MUSCLE ATROPHY; MUSCLE DISEASE; MUSCLE WEAKNESS; MYALGIA; NEMALINE MYOPATHY; PLASMA CELL DYSCRASIA; REVIEW; SPORADIC LATE ONSET NEMALINE MYOPATHY; META ANALYSIS; PATHOLOGY; PATHOPHYSIOLOGY; SKELETAL MUSCLE; STEM CELL TRANSPLANTATION;

HUMANS; MONOCLONAL GAMMOPATHY OF UNDETERMINED SIGNIFICANCE; MUSCLE, SKELETAL; MYOPATHIES, NEMALINE; STEM CELL TRANSPLANTATION;

EID: 85021824511     PISSN: 13507540     EISSN: 14736551     Source Type: Journal    
DOI: 10.1097/WCO.0000000000000477     Document Type: Review

References (53)

1. Shy GM, Engel WK, Somers JE, et al. Nemaline myopathy. A new congenital myopathy. Brain 1963; 86:793-810.
2. Miyatake S, Mitsuhashi S, Hayashi YK, et al. Biallelic mutations in MYPN, encoding myopalladin, are associated with childhood-onset, slowly progressive nemaline myopathy. Am J Hum Genet 2017; 100:169-178.
3. Engel AG. Late-onset rod myopathy (a new syndrome?): light and electron microscopic observations in two cases. Mayo Clin Proc 1966; 41:713-741.
4. Engel WK, Oberc MA. Abundant nuclear rods in adult-onset rod disease. J Neuropathol Exp Neurol 1975; 34:119-132.
5. Seitz RJ, Toyka KV, Wechsler W. Adult-onset mixed myopathy with nemaline rods, minicores, and central cores: a muscle disorder mimicking polymyositis. J Neurol 1984; 231:103-108.
6. Eymard B, Brouet JC, Collin H, et al. Late-onset rod myopathy associated with monoclonal gammopathy. Neuromuscul Disord 1993; 3:557-560.
7. Deconinck N, Laterre EC, Van den Bergh PY. Adult-onset nemaline myopathy and monoclonal gammopathy: a case report. Acta Neurol Belg 2000; 100:34-40.
8. Chahin N, Selcen D, Engel AG. Sporadic late onset nemaline myopathy. Neurology 2005; 65:1158-1164.
9. Keller CE, Hays AP, Rowland LP, et al. Adult-onset nemaline myopathy and monoclonal gammopathy. Arch Neurol 2006; 63:132-134.
10. Benveniste O, Laforet P, Dubourg O, et al. Stem cell transplantation in a patient with late-onset nemaline myopathy and gammopathy. Neurology 2008; 71:531-532.
11. Voermans NC, Minnema M, Lammens M, et al. Sporadic late-onset nemaline myopathy effectively treated by melphalan and stem cell transplant. Neurology 2008; 71:532-534.
12. Irodenko VS, Lee HS, de Armond SJ, et al. Adult nemaline myopathy with trabecular muscle fibers. Muscle Nerve 2009; 39:871-875.
13. Novy J, Rosselet A, Spertini O, et al. Muscle Nerve 2010; 41:286-287.
14. Milone M, Katz A, Amato AA, et al. Sporadic late onset nemaline myopathy responsive to IVIg and immunotherapy. Muscle Nerve 2010; 41:272-276.
15. Doppler K, Knop S, Einsele H, et al. Sporadic late-onset nemaline myopathy and immunoglobulin deposition disease. Muscle Nerve 2013; 48:983-988.
16. Voermans NC, Benveniste O, Minnema MC, et al. Sporadic late-onset nemaline myopathy with MGUS: long-term follow-up after melphalan and SCT. Neurology 2014; 83:2133-2139.
17. Maeda MH, Ohta H, Izutsu K, et al. Sporadic late-onset nemaline myopathy as a rare cause of slowly progressive muscle weakness with young adult onset. Muscle Nerve 2015; 51:772-774.
18. Montagnese F, Portaro S, Musumeci O, et al. Sporadic late-onset nemaline myopathy in a woman with multiple myeloma successfully treated with lenalidomide/dexamethasone. Muscle Nerve 2015; 51:934-935.
19. Belkhribchia MR, Tazi I, Louhab N, et al. Autologous stem cell transplantation in a patient with sporadic late-onset nemaline myopathy and monoclonal gammopathy: first Moroccan experience. Presse Med 2017; 46:122-125.
20. Sarullo FM, Vitale G, Di Franco A, et al. Nemaline myopathy and heart failure: role of ivabradine; a case report. BMC Cardiovasc Disord 2015; 15:5.
21. Mizuno Y, Mori-Yoshimura M, Okamoto T, et al. Two cases of sporadic late onset nemaline myopathy effectively treated with immunotherapy. Rinsho Shinkeigaku 2016; 56:605-611.
22. Belhomme N, Maamar A, Le Gallou T, et al. Rare myopathy associated to MGUS, causing heart failure and responding to chemotherapy. Ann Hematol 2017; 96:695-696.
23. Dalakas MC, Pezeshkpour GH, Flaherty M. Progressive nemaline (rod) myopathy associated with HIV infection. N Engl J Med 1987; 317:1602-1603.
24. Gonzales MF, Olney RK, So YT, et al. Subacute structural myopathy associated with human immunodeficiency virus infection. Arch Neurol 1988; 45:585-587.
25. Simpson DM, Bender AN. Human immunodeficiency virus-associated myopathy: analysis of 11 patients. Ann Neurol 1988; 24:79-84.
26. Cabello A, Martinez-Martin P, Gutierrez-Rivas E, Madero S. Myopathy with nemaline structures associated with HIV infection. J Neurol 1990; 237:64-66.
27. Dwyer BA, Mayer RF, Lee SC. Progressive nemaline (rod) myopathy as a presentation of human immunodeficiency virus infection. Arch Neurol 1992; 49:440.
28. Maytal J, Horowitz S, Lipper S, et al. Progressive nemaline rod myopathy in a woman coinfected with HIV-1 and HTLV-2. Mt Sinai J Med 1993; 60:242-246.
29. Feinberg DM, Spiro AJ, Weidenheim KM. Distinct light microscopic changes in human immunodeficiency virus-associated nemaline myopathy. Neurology 1998; 50:529-531.
30. Madonia P,Wilson J, BicanO, et al. HIV, rods, and the muscles-a discussion about HIV-associated nemaline rod myopathy. J La State Med Soc 2012; 164:320-323.
31. Lomen-Hoerth C, Simmons ML, Dearmond SJ, et al. Adult-onset nemaline myopathy: another cause of dropped head. Muscle Nerve 1999; 22:1146-1150.
32. Bradwell AR, Carr-Smith HD, Mead GP, et al. Highly sensitive, automated immunoassay for immunoglobulin free light chains in serum and urine. Clin Chem 2001; 47:673-680.
33. Dispenzieri A, Kyle R, Merlini G, et al. International Myeloma Working Group guidelines for serum-free light chain analysis in multiple myeloma and related disorders. Leukemia 2009; 23:215-224.
34. Katzmann JA, Dispenzieri A, Kyle RA, et al. Elimination of the need for urine studies in the screening algorithm for monoclonal gammopathies by using serum immunofixation and free light chain assays. Mayo Clin Proc 2006; 81:1575-1578.
35. Kimura J. Electrodiagnosis in diseases of nerve and muscle: principles and practice. 3rd ed. New York: Oxford University Press; 2001; 797-802.
36. Ryan MM, Ilkovski B, Strickland CD, et al. Clinical course correlates poorly with muscle pathology in nemaline myopathy. Neurology 2003; 60:665-673.
37. Cape CA, Johnson WW, Pitner SE. Nemaline structures in polymyositis. A nonspecific pathological reaction of skeletal muscles. Neurology 1970; 20:494-502.
38. Martinez AJ, Hooshmand H, Faris AA. Acute alcoholic myopathy. Enzyme histochemistry and electron microscopic findings. J Neurol Sci 1973; 20:245-252.
39. Reyes MG, Tal A, Abrahamson D, et al. Nemaline myopathy in an adult with primary hypothyroidism. Can J Neurol Sci 1986; 13:117-119.
40. Konno H, Iwasaki Y, Yamamoto T, et al. Nemaline bodies in spinal progressive muscular atrophy. An autopsy case. Acta Neuropathol 1987; 74:84-88.
41. Kimura M, Furuta T, Hiruma S, et al. Nemaline bodies of skeletal muscle fibers of the neck in a case of pharyngeal cancer. Pathol Int 1997; 47:256-259.
42. Sun AP, Ohtsuki Y, Yano T, et al. Typical nemaline bodies presenting in a patient with polymyositis. Med Electron Microsc 2002; 35:167-172.
43. Buxbaum JN, Chuba JV, Hellman GC, et al. Monoclonal immunoglobulin deposition disease: light chain and light and heavy chain deposition diseases and their relation to light chain amyloidosis. Clinical features, immunopathology, and molecular analysis. Ann Intern Med 1990; 112:455-464.
44. Ronco PM, Alyanakian MA, Mougenot B, et al. Light chain deposition disease: a model of glomerulosclerosis defined at the molecular level. J Am Soc Nephrol 2001; 12:1558-1565.
45. Ronco P, Plaisier E, Mougenot B, et al. Immunoglobulin light (heavy)-chain deposition disease: from molecular medicine to pathophysiology-driven therapy. Clin J Am Soc Nephrol 2006; 1:1342-1350.
46. Weichman K, Dember LM, Prokaeva T, et al. Clinical and molecular characteristics of patients with nonamyloid light chain deposition disorders, and outcome following treatment with high-dose melphalan and autologous stem cell transplantation. Bone Marrow Transplant 2006; 38:339-343.
47. Faa G, Van Eyken P, De Vos R, et al. Light chain deposition disease of the liver associated with AL-type amyloidosis and severe cholestasis: a case report and literature review. J Hepatol 1991; 12:75-82.
48. Kasahara N, Tamura H, Matsumura O, et al. An autopsy case of light-chain deposition disease. Intern Med 1994; 33:216-221.
49. Jantunen E, Itä lä M, Lehtinen T, et al. Early treatment-related mortality in adult autologous stem cell transplant recipients: a nation-wide survey of 1482 transplanted patients. Eur J Haematol 2006; 76:245-250.
50. Skinner M, Sanchorawala V, Seldin DC, et al. High-dose melphalan and autologous stem-cell transplantation in patients with AL amyloidosis: an 8-year study. Ann Intern Med 2004; 140:85-93.
51. Gertz MA, Lacy MQ, Gastineau DA, et al. Blood stem cell transplantation as therapy for primary systemic amyloidosis (AL). Bone MarrowTransplant 2000; 26:963-969.
52. Sonneveld P, Avet-Loiseau H, Lonial S, et al. Treatment of multiple myeloma with high-risk cytogenetics: a consensus of the International Myeloma Working Group. Blood 2016; 127:2955-2962.
53. Merlini G, Stone MJ. Dangerous small B-cell clones. Blood 2006; 108:2520-2530.