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Drug Profile

Amifampridine for the treatment of Lambert-Eaton myasthenic syndrome

Pages 991-1007 | Received 27 Jun 2019, Accepted 17 Sep 2019, Published online: 30 Sep 2019

References

  • Wirtz PW, van Dijk JG, van Doorn PA, et al. The epidemiology of the Lambert-Eaton myasthenic syndrome in the Netherlands. Neurology. 2004;63:397–398.
  • Sanders DB. Lambert-Eaton myasthenic syndrome: diagnosis and treatment. Ann N Y Acad Sci. 2003;998:500–508.
  • Lennon VA, Kryzer TJ, Griesmann GE, et al. Calcium- channel antibodies in the Lambert–Eaton syndrome and other paraneoplastic syndromes. NEJM. 1995;332:1467–1474.
  • O’Neill JH, Murray NM, Newsom-Davis J. The Lambert-Eaton myasthenic syndrome. A review of 50 cases. Brain. 1988;111(Pt 3):577–596.
  • Oh SJ, Kurokawa K, Claussen GC, et al. Electrophysiological diagnostic criteria of Lambert-Eaton myasthenic syndrome. Muscle Nerve. 2005;32:515–520.
  • Lambert EH, Elmqvist D. Quantal components of end-plate potentials in the myasthenic syndrome. Ann N Y Acad Sci. 1971;183:183–199.
  • Lambert E, Rooke E. Myasthenic state and lung cancer. In: Brain L, Norris F Jr, editors. The remote effects of cancer on the nervous system. New York,NY: Grune & Stratton; 1965. p. 67–80.
  • Gutmann L, Phillips LH 2nd, Gutmann L. Tredns in the association of Lambert-Eaton myasthenic syndrome with carcinoma. Neurology. 1992;42:848–850.
  • Titulaer MJ, Wirtz PW, Willems LN, et al. Screening for small-cell lung cancer: a follow-upstudy of patients with Lambert-Eaton myasthenicsyndrome. J Clin Oncol. 2008;26:4276–4281.
  • Odabasi Z, Demirci M, D.S. Kim DS, et al. Postexercise facilitation of reflexes is not common in Lambert–Eaton myasthenic syndrome. Neurology. 2002;59:1085–1087.
  • Oh SJ. Distinguishing features of the repetitive nerve stimulation test between Lambert–Eaton myasthenic syndrome and myasthenia gravis, 50-year reappraisal. J Clin Neuromuscular Dis. 2017;19:66–75.
  • Hatanaka Y, Oh SJ. Ten-second exercise is superior to 30-second exercise for post-exercise facilitation in diagnosing Lambert-Eaton myasthenic syndrome. Muscle Nerve. 2008;37:572.
  • Motomura M, Lang B, Johnston I, et al. Incidence of serum anti-P/O-type and anti-N-type calcium channel autoantibodies in the Lambert–Eaton myasthenic syndrome. J Neurol Sci. 1997;147:35–42.
  • Di Lorenzo R, Karin Mente K, Li J, et al. Low specificity of voltage-gated calcium channel antibodies in Lambert–Eaton myasthenic syndrome: a call for caution. J Neurol. 2018;265:2114–2119.
  • Zalewski NL, Lennon VA, Lachance DH, et al. P/Q- and N-type calcium-channel antibodies: oncological, neurological, and serological accompaniments. Muscle Nerve. 2016;54:220–227.
  • Oh SJ, Hatanaka Y, Claussen GC, et al. Electrophysiological differences in seropositive and seronegative Lambert-Eaton myasthenic syndrome. Muscle Nerve. 2007;35:178–183.
  • Sabater L, Titulaer M, Saiz A, et al. SOX1 antibodies are markers of paraneoplastic Lambert–eaton myasthenic syndrome. Neurology. 2008;70:924–928.
  • Oh SJ. Treatment and management of disorders of the neuromuscular junction. In: Bertorini TE, editor. Neuromuscular disorders. Treatment and management. Philadelphia, PA: Sanders Elsevier; 2011. p. 307–342.
  • Bain PG, Motomura M, Newsome-Davis J, et al. Effects of intravenous immunoglobulin on muscle weakness and calcium-channel autoantibodies in the Lambert-Eaton myasthenic syndrome. Neurology. 1996;47:678–683.
  • Kesner VG, Oh SJ, Dimacheke MM, et al. Lambert-Eaton myasthenic syndrome. Neurol Clin. 2018;36:379–394.
  • Newsom-Davis J. A treatment algorithm for Lambert-Eaton myasthenic syndrome. Ann NY Acad Sci. 1998;841:817–822.
  • Chalk CH, Murray NM, Newsom-Davis J, et al. Response of the Lambert-Eaton myasthenic syndrome to treatment of associated small-cell lung carcinoma. Neurology. 1990;40:1552–1556.
  • Maddisson P, Newsome-Davis J, Mills KR, et al. Favorable prognosis in Lambert-Eaton myasthenic syndrome and small-cell lung carcinoma. Lancet. 1999;353:117–118.
  • Maddison P, Gozzard P, Grainge MJ, et al. Long-term survival in paraneoplastic Lambert-Eaton myasthenic syndrome. Neurology. 2017;88:1334–1339.
  • Oh SJ, Cho H. Edrophonium responsiveness not necessarily diagnostic of myasthenia gravis. Muscle Nerve. 1990;13:187–191.
  • Wirtz PW, Verschuuren JJ, van Diik JG, et al. Efficacy of 3,4-diaminopyridine and pyridostgmine in the treatment of Lambert-Eaton myasthenic syndrome: a randomized, double-bldind, placebo-controlled crossover study. Clin Pharmacol Ther. 2009;86:44–48.
  • Verschuuren JJ, Wirtz PW, Titulaer MJ, et al. Available treatment options for the management of Lambert-Eaton myasthenic syndrome. Expert Opin Pharmacother. 2006;7:1323–1336.
  • Oh SJ, Kim DS, Kwon KH, et al. Wide spectrum of symptomatic treatment in LEMS. Ann NY Acad Sci. 1998;841:827–831.
  • Lundh H, Nilsson O, Rosen I. Treatment of Lambert-Eaton syndrome: 3,4-diaminopyridine and pyridostigmine. Neurology. 1984;34:1324–1330.
  • Lundh H, Nilsson O, Rosen I, et al. Practical aspects of3,4-diaminopyridine treatment of the Lambert-Eaton myasthenic syndrome. Acta Neurol Scand. 1993;88:136–140.
  • Oh SJ, Kim DS, Head T, et al. Low-dose guanidine and pyridostigmine: relatively safe and effective long-term symptomatic therapy in Lambert-Eaton myasthenic syndrome. Muscle Nerve. 1997;20:1146–1152.
  • Tim RW, Massey JM, Sanders DB. Lambert-Eaton myasthenic syndrome: electrodiagnostic findings and response to treatment. Neurology. 2000;54:2176–2178.
  • Tim RW, Massey JM, Sanders DB. Lambert-Eaton myasthenic syndrome(LEMS). Clinical and electrodiagnostic features and response to therapy in 59 patients. Ann N Y Acad Sci. 1998;841:823–826.
  • Tarr TB, Malick W, Liang M, et al. Evaluation of a novel calcium channel agonist for therapeutic potential in Lambert-Eaton myasthenic syndrome. J Neurosci. 2013;33:10559–10567.
  • Tarr TB, Lacomis D, Redell SW, et al. Complete reversal of Lambert-Eaton myashthenic syndrome synaptic impairment by the combined use of a K+ channel blocker and a Ca2+ channel agnosit. J Physiol. 2014;592:3687–3697.
  • Thomsen RH, Wilson DF. Effects of 4-aminopyridine and 3,4-diaminopyridine on transmitter release at the neuromuscular junction. J Pharmacol Exp Ther. 1983;227:260–265.
  • Oh SJ, Sieb JP. Update on amifampridine as a drug of choice in Lambert-Eaton myasthenic syndrome. US Neurol. 2014;10:83–89.
  • Oh SJ, Kim KW. Guanidine hydrochloride in the Eaton-Lambert syndrome. Neurology. 1973;23:1084–1090.
  • Lunch H, Nilsson O, Rosen I. 4-Aminopyridine – a new drug tested in the treatment of Eaton-Lambert syndrome. J Neurol Neurosurg Psychiatry. 1977;40:1109–1112.
  • Raust JA, Goulay-Dufay S, Le Hoang MD, et al. Stability studies of ionised and non-ionised 3,4-diaminopyridine: hypothesis of degradation pathways and chemical structure of degradation products. J Pharm Biomed Anal. 2007;43:83–88.
  • Lindquist S, Strangel M. Update on treatment options for Lambert-Eason myasthenic syndrome; focus on use of amifampridine. Neuropsych Dis Treat. 2011;7:341–349.
  • Lundh H, Onilsson O, Rosen I. Novel drug of choice in Eaton-Lambert Syndrome. J Neurol Neurosurg Psychiatry. 1983;46:684–687.
  • Sanders DB. 3,4-diaminopyrdine (DAP) in the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS). Ann NY Acad Sci. 1998;841:811–816.
  • Funck-Bretano C. Relative bioavailability study of 3,4-DAP administered as a salt or a base. DAPSEL study. Clin Study Rep. 2006 Feb.
  • Oh SJ. Myasthenia gravis Lambert-Eaton myasthenic syndrome. Muscle Nerve. 2016;53:20–26.
  • Smith AG, Wald J. Acute ventilatory failure in Lambert-Eaton myasthenic syndrome and its response to 3,4-diaminopyridine. Neurology. 1996;46:1143–1145.
  • Brueck M, Vogel S, Waegr S, et al. Lambert-Easton myasthenic syndrome with acute respiratory failure associated with small cell lung cancer. Dtsch Med Wochenschr. 2004;129:193–196.
  • McEvoy KM, Windebank AJ, Daube JR. Low PA:3,4-diaminopyridine in the treatment of Lambert-Eaton myasthenic syndrome. N Engl J Med. 1989;321:1567–1571.
  • Jost WH, Mielke U, Forrett-Kaminsky MC, et al. Tritment au long cours du syndrome de Lambert-Eaton paar la 3,4 diaminopyridine. Rev Neurol. 1992;148:276–279.
  • Satoh K, Motomura M, Suzu H, et al. Neurogenic bladder in Lambert-Eaton myasthenic syndrome and its response to 3,4-diaminopyridine. J Neurol Sci. 2013;183:1–4.
  • Nagamura R, Yabe I, Takahashi I, et al. A restrospective study of the effects of 3,4-daiaminopridine treatment in Lambert-Eaton myasthenic syndrome. Rinsho Sinkeigaku. 2018;58:83–87.
  • MK L, Sunwoo IN, Kim SM. 3,4-Diaminopyridine for the treatment of myasthenia gravis with electrophysiological patterns of Lambert-Eaton myasthenic syndrome. J Clin Neurosci. 2018;50:194–198.
  • Portaro BT, Sinicropi S, Cacciola A, et al. Five years experience on 3,4-diaminopyridine phosphate in Lambert–Eaton syndrome. Case reports. Medicine (Baltimore). 2017 Sep;96(38):e7839. doi:10.1097/MD.0000000000007839. PMID:2893082.
  • Rattananan W, Alsharabati M, Oh SJ. Ocular LEMS or MLOS. Muscle Nerve. 2016;54:681–682.
  • Mantegazza R, Meisel A, Joern P, et al. The European LEMS registry: baseline demographics and treatment approaches. Neurol Therapy. 2015;4:105–124.
  • Murray NM, Newsom-Davis J, Karni Y. Oral 3,4-diaminopyridine in the treatment of the Lambert-Eaton myasthenic syndrom (LEMS) (abstract). J Neurol Neurosurg Psychiatry. 1984;47:1052.
  • Sanders DB, Howard JF, Massey JM. 3,4-Diaminopyridine in Lambert-Eaton myasthenic syndrome and myasthenia gravis. Ann NY Acad Sci. 1993;681:588–590.
  • Sanders DB, Massey J, Sanders LL, et al. A randomized trial of 3,4-diaminopyridine in Lambert-Eaton myasthenic syndrome. Neurology. 2000;54:603–607.
  • Oh SJ, Claussen GG, Hatanaka Y, et al. 3,4 Diaminopyridine is more effective than placebo in a randomized, double-blind, cross-over drug study in LEMS. Muscle Nerve. 2009;40:795–800.
  • Sanders DB, Juel VC, Harti Y, et al. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018;57:561–568.
  • Oh SJ, Shcherbakova N, Kostera-Pruszczyk A, et al. Amifampridine phosphate (FirdapseR) is effective and safe in a phase 3 clinical trial in LEMS. Muscle Nerve. 2016;53(5):717–725.
  • Shieh P, Sharma K, Kohrman B, et al. Amifampridine phosphate (Firdapse) is effective in a confirmatory phase 3 clinical trial in LEMS. J Clin Neuromuscul Dis. 2019;20:111–119.
  • Boerma CE, Rommes JH, Van Leeuwen RB, et al. Cardiac arrest following an iatrogenic 3,4-diaminopyridine intoxication in a patient with Lambert-Eaton myasthenic syndrome. J Toxicol Clin Toxicol. 1995;33(3):249–251.
  • Flet L, Polard E, Guillard O, et al. 3,4-diaminopyridine safety in clinical practice: an observational,retrospective cohort study. J Neurol. 2010;257:937–946.
  • Kanzato N, Moromura M, Suehara M, et al. Lambert-Eaton myasthenic syndrome with ophthalmparesis and pseudoblepharospasm. Muscle Nerve. 1999;22:1727–1730.
  • Quartel A, Tuberville S, Lounsury D. Current therapy for Lambert-Eaton myasthenic syndrome: development of 3,4-diaminopyridine phosphate salt as first-line symptomatic treatment. CMRO. 2010;26:1363–1375.
  • Pelufo-Pellicer A, Monte-Boquet E, Roma-Sanchez E, et al. Fetal exposure to 3,4-diaminopyridine in a pregnant woman with a congenital myasthenic syndrome. Ann Pharmacother. 2006;40:762–766.
  • Lundh H, Nisson O, Rosen I. Improvement in neuromuscular transmission in myasthenia gravis by 3,4-diaminopyridine. Eur Arch Psychiatry Neurol Sci. 1985;234:374–377.
  • Hatanaka Y, Hemmi S, Morgan MB, et al. Nonresponsiveness to anticholinesterase agents in patients with MuSK-antibody positive MG. Neurology. 2005;65:1508–1509.
  • Skjei KI, Lennon VA, Kuntz NL. Muscle specific kinase autoimmune myasthenia gravis in children: a case series. Neuromuscular Disord. 2013;23:874–882.
  • Evoli A, Alboini PE, Damao V, et al. 3,4-Diaminopyridine may improve myasthenia gravis with MuSK antibodies. Neurology. 2016;86:1070–1107.
  • Bonnanno S, Pasanisi MB, Frangiamore R, et al. Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study. SAGE Open Med. 2018;6:1–9.
  • Palace J, Wiles CM, Newsom-Davis J. 3,4-Diaminopyridine in the treatment of congenital (hereditary) myasthenic syndrome. J Neurol Neurosurg Psychiatry. 1991;54:1069–1072.
  • Anlar B, Varli K, Ozdirim E, et al. 3,4-Diaminopyridine in childhood myasthenia: double-blind placebo-controlled trial. J Child Neurol. 1996;11:458–461.
  • Finlayson S, Palace J, Belaya K, et al. Clinical features of congenital myasthenic syndrome due to mutations in DPAGT1. J Neurol Neurosurg Psychiatry. 2013;84:1119–1125.
  • Verma S, Mazell SN, Shah DA. Amifampridine phosphate in congenital myasthenic syndrome. Muscle Nerve. 2016;54:809–810.
  • Natera-de Benito D, Bestué M, Vilchez JJ, et al. Long-term follow-up in patients with congenital myasthenic syndrome due to RAPSN mutations. Neuromuscul Disord. 2016;26:153–159.
  • Whittaker RG, Herrmann DN, Bansagi B, et al. Electrophysiologic features of SYT2 mutations causing a treatable neuromuscular syndrome. Neurology. 2015;85:1964–1971.
  • Nishikawa A, Mori-Yoshimura M, Segawa K, et al. Beneficial effects of 3,4-diaminopyridine in a 26-year-old woman with DOK7 congenital myasthenic syndrome who was originally diagnosed with facioscapulohumeral dystrophy. Rinsho Shinkeigaku. 2014;54:561–564.
  • Palace J, Lashley D, Bailey S, et al. Clinical features in a series of fast channel congenital myasthenia syndrome. Neuromuscul Disord. 2013;22:112–117.
  • Banwell BL, Ohno K, Sieb JP, et al. AG: novel truncating RAPSN mutations causing congenital myasthenic syndrome responsive to 3,4-diaminopyridine. Neuromuscul Disord. 2004;14:202–207.
  • Dock M, Ben Ali A, Karras A, et al. Treatment of severe botulism with 3,4-diaminopyridine. Presse Med. 2002;31:601–602.
  • Friggeri A, Marcon F, Marciniak S, et al. 3,4-Diaminopyridine may improve neuromuscular block during botulism. Crit Care. 2013;17:449.
  • Skie GO, Apostolski S, Evoli, et al. Guidelines for the treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol. 2006;13:682–690.
  • De Wilde SD, de Jong GH, Jong MGH, et al. The possibility of obtaining marketing authorization of orphan pharmaceurtical compounding preparations: 3,4-DAP for Lambert-Eaton myasthenic syndrome. Eur J Pharm Sci. 2018;114:24–29.
  • Burns TM, Smith GA, Allen JA, et al. Editorial by concerned physicians: unintended effect of the orphan drug act on the potential cost of 3,4-diaminopyridine. Muscle Nerve. 2016;53:165–168.
  • Sadeh M, River Y, Argov Z. Stimulated single-fiber electromyography in Lambert-Eaton myasthenic syndrome before and after 3,4-diaminopyridine. Muscle Nerve. 1997;20:735–739.
  • Abenroth DC, Smith AG, Greenlee JE, et al. Lambert-Eaton myasthenic syndrome: epidemiology and therapeutic response in the national veterans affairs populationl. Muscle Nerve. 2017;56:421–426.
  • Hyun JW, Kim SH, Jeong IH, et al. Successful treatment of Lambert-Eaton myasythenic syndrome in a small cell lung cancer patient using 3,4-diaminopyridine: A case report. Oncol Lett. 2016;11:1933–1935.
  • Chan K, Chang RS, Lau VW, et al. Palliative care for patient with Lambert-Eaton myasthenic syndrome: role of 3,4 diaminopyridine. Ann Palliat Med. 2016;5:311–314.
  • Alboini PE, Damato V, Iorio R, et al. Myasthenia gravis with presynaptic neurophysiological signs. Two case reports and literature review. Neuromuscular Disord. 2015;25:646–650.

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