Romiplostim: A novel c-Mpl/CD110 receptor ligand for the management of idiopathic thrombocytopenic purpura

H. Patel, N. Patel, A. Vyas, M. Patel, S. Pandey

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

Idiopathic Thrombocytopenic Purpura (ITP) is an autoimmune disease characterised predominantly by an antibody mediated destruction of platelets. Few treatment options are available for ITP like corticosteroids, IV immunoglobulins, splenectomy, rituximab, danazole and cyclophosphamide. All these treatment options work by reducing the destruction of platelets by antibody. However recent evidence suggests that production of platelet is reduced in 75% of thrombocytopenic patient with ITP. Romiplostim is a novel thrombopoetin receptor agonist approved by USFDA in August 2008 that increases the platelet count in ITP patients unresponsive to corticosteroids, immunoglobulins or splenectomy. Romiplostim is not currently available in India. The addition of Romiplostim in the treatment options for ITP will surely improve the outcomes in ITP patients.
Original languageEnglish
Pages (from-to)1690-1696
Number of pages7
JournalJournal of Clinical and Diagnostic Research
Volume3
Issue number4
Publication statusPublished - 2009

Fingerprint

Idiopathic Thrombocytopenic Purpura
Platelets
Ligands
Adrenal Cortex Hormones
Blood Platelets
Splenectomy
Antibodies
Intravenous Immunoglobulins
Cyclophosphamide
Immunoglobulins
United States Food and Drug Administration
Platelet Count
Autoimmune Diseases
romiplostim
India
Therapeutics

Cite this

Patel, H. ; Patel, N. ; Vyas, A. ; Patel, M. ; Pandey, S. / Romiplostim: A novel c-Mpl/CD110 receptor ligand for the management of idiopathic thrombocytopenic purpura. In: Journal of Clinical and Diagnostic Research. 2009 ; Vol. 3, No. 4. pp. 1690-1696.
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title = "Romiplostim: A novel c-Mpl/CD110 receptor ligand for the management of idiopathic thrombocytopenic purpura",
abstract = "Idiopathic Thrombocytopenic Purpura (ITP) is an autoimmune disease characterised predominantly by an antibody mediated destruction of platelets. Few treatment options are available for ITP like corticosteroids, IV immunoglobulins, splenectomy, rituximab, danazole and cyclophosphamide. All these treatment options work by reducing the destruction of platelets by antibody. However recent evidence suggests that production of platelet is reduced in 75{\%} of thrombocytopenic patient with ITP. Romiplostim is a novel thrombopoetin receptor agonist approved by USFDA in August 2008 that increases the platelet count in ITP patients unresponsive to corticosteroids, immunoglobulins or splenectomy. Romiplostim is not currently available in India. The addition of Romiplostim in the treatment options for ITP will surely improve the outcomes in ITP patients.",
author = "H. Patel and N. Patel and A. Vyas and M. Patel and S. Pandey",
note = "Cited By :1 Export Date: 10 November 2017 Correspondence Address: Patel, H.223 Raman Block, Udupi, Karnataka 576104, India; email: hiren379@gmail.com Chemicals/CAS: cyclophosphamide, 50-18-0; danazol, 17230-88-5; immunoglobulin, 9007-83-4; rituximab, 174722-31-7; romiplostim, 267639-76-9, 847595-08-8 References: George, J.N., El-Harake, M.A., Raskob, G.E., Chronic idiopathic thrombocytopenic purpura (1994) N Engl J Med, 331 (18), pp. 1207-1211. , Nov 3;; Karpatkin, S., Autoimmune (idiopathic) thrombocytopenic purpura (1997) Lancet, 349 (9064), pp. 1531-1536. , May 24;; Cohen, Y.C., Djulbegovic, B., Shamai-Lubovitz, O., Mozes, B., The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts (2000) Arch Intern Med, 160 (11), pp. 1630-1638. , Jun 12;; George, J.N., Kojouri, K., Perdue, J.J., Vesely, S.K., Management of patients with chronic, refractory idiopathic thrombocytopenic purpura (2000) Semin Hematol, 37 (3), pp. 290-298. , Jul;; George, J.N., Woolf, S.H., Raskob, G.E., Wasser, J.S., Aledort, L.M., Ballem, P.J., Idiopathic thrombocytopenic purpura: A practice guideline developed by explicit methods for the American Society of Hematology (1996) Blood, 88 (1), pp. 3-40. , Jul 1;; Scaradavou, A., Woo, B., Woloski, B.M., Cunningham-Rundles, S., Ettinger, L.J., Aledort, L.M., Intravenous anti-D treatment of immune thrombocytopenic purpura: Experience in 272 patients (1997) Blood, 89 (8), pp. 2689-2700. , Apr 15;; Stasi, R., Provan, D., Management of immune thrombocytopenic purpura in adults (2004) Mayo Clin Proc, 79 (4), pp. 504-522. , Apr;; McMillan, R., Wang, L., Tomer, A., Nichol, J., Pistillo, J., Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP (2004) Blood, 103 (4), pp. 1364-1369. , Oct 23;; Heyns, A.D., Lotter, M.G., Badenhorst, P.N., Kock, F.D., Pieters, H., Herbst, C., Kinetics and sites of destruction of 111Indium-oxine-labeled platelets in idiopathic thrombocytopenic purpura: A quantitative study (1982) Am J Hematol, 12 (2), pp. 167-177; Ballem, P.J., Segal, G.M., Stratton, J.R., Gernsheimer, T., Adamson, J.W., Slichter, S.J., Mechanisms of thrombocytopenia in chronic autoimmune thrombocytopenic purpura. Evidence of both impaired platelet production and increased platelet clearance (1987) J Clin Invest, 80 (1), pp. 33-40. , Jul;; Nichol, J.L., Thrombopoietin levels after chemotherapy and in naturally occurring human diseases (1998) Curr Opin Hematol, 5 (3), pp. 203-208. , May;; Kosugi, S., Kurata, Y., Tomiyama, Y., Circulating thrombopoietin level in chronic immune thrombocytopenic purpura (1996) Br J Haematol, 93 (3), pp. 704-706; Emmons, R.V., Reid, D.M., Cohen, R.L., Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction (1996) Blood, 87, pp. 4068-4071; Houwerzijl, E.J., Blom, N.R., van der Want, J.J., Esselink, M.T., Koornstra, J.J., Smit, J.W., Ultrastructural study shows morphologic features of apoptosis and para-apoptosis in megakaryocytes from patients with idiopathic thrombocytopenic purpura (2004) Blood, 103 (2), pp. 500-506. , Jan 15;; Kuter, D.J., Bussel, J.B., Lyons, R.M., Pullarkat, V., Gernsheimer, T.B., Senecal, F.M., Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: A double-blind randomized controlled trial (2002) Lancet, 371, pp. 395-403. , Feb 2;; Kuter, D.J., New thrombopoietic growth factors (2007) Blood, 109 (11), pp. 4607-4616. , Jun 1;; Bussel, J.B., Kuter, D.J., George, J.N., Mcmillan, R., Aledort, L.M., Conklin, G.T., AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP (2006) N Engl J Med, 355 (16), pp. 1672-1681. , Oct 19;; Bussel JB, Kutter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood [serial online ] 2008 Nov 3 [cited 2009 Jan 28]: Available from: URL: http://bloodjournal.hematologylibrary.org/ cgi/content/abstract/blood-2008-04-150078v1; Highlights of prescribing information of romiplostim, , http://www.fda.gov/cder/foi/label/2008/125268lbl.pdf, Online, 2008 Aug [cited 2009 Jan 28, 1,2,6,8. Available from: URL; Peetersk, K., Stassen, J.M., Collen, D., Geet, C.V., Freson, K., Emerging treatments for thrombocytopenia: Increasing platelet production (2008) Drug discovery today, 13 (17-18), pp. 798-806. , Sep;; Wang, B., Nichol, J.L., Sullivan, J.T., Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor (2004) Clin Pharmacol Ther, 76 (6), pp. 628-638; Stasi, R., Evangelista, M.L., Amadori, S., Novel thrombopoietic agents: A review of their use in idiopathic thrombocytopenia purpura (2008) Drugs, 68 (7), pp. 901-912; Methia, N., Louache, F., Vainchenker, W., Wendling, F., Oligodeoxynucleotides antisense to the proto-oncogene c-mpl specifically inhibit in vitro megakaryopoiesis (1993) Blood, 82 (5), pp. 1395-1401. , Sep 1;; Lodish, H., Berk, A., Kaiser, C.A., Krieger, M., Scott, M.P., Bretscher, A., (2008) Molecular cell biology, pp. 672-676. , 6th ed. New York: W. H. Freeman and Company;, P; Clinical Pharmacology and biopharmaceutics Reviews of romiplostim. Food and drug Administration, , http://www.fda.gov/cder/foi/nda/2008/125268s000_ClinPharmR.pdf, online, cited 2009 Jan 28];Available from: URL; Kumagi, Y., Fujita, T., Ozaki, M., Sahashi, K., Ohkura, M., Ohtsu, T., Pharmacodynamics and pharmacokinetics of AMJ531, a Thrombopoesis Stimulating peptibody, in Healthy Japanese Subjects: A Randomized, Placebo Controlled study (2007) J Clin Pharmacol, 47, pp. 1489-1497; Newland, A., Caulier, M.T., Kappers, K.M., An open label, unit dose finding study of AMG 531, a novel thrombopoesis stimulating peptibody in patient with ITP (2006) Br J Haematol, 135, pp. 547-553; George J, Bussel JB, Lyons RM, Pullarkat V, Redner R, Terreu D. et al. Self injection of Romiplostim by patients with chronic immune thrombocytopenic Purpura(ITP). Blood [serial online] 2008 Dec [cited 2009 Jan 29]; 112: Available from : URL: http://www.cancercaresouthtexas.com/docs/ SelfInjection{\%}20of{\%}20Romiplostim{\%}20by{\%}20 Patients{\%}20with{\%}20Chronic{\%}20Immune{\%}20Throbocytopeni.pdf; Cines, D.B., Yasothan, U., Kirkpatrick, P., Romiplostim, (2008) Nature Review Drug Discovery, pp. 887-888. , Nov 7; Oncologic drug advisory committee. FDA advisory committee briefing document on romiplostim [Online]. 2008 Mar [cited 2009 Jan 25]; 3,6-9. Available from: URL: http://www.fda.gov/ohrms/dockets/AC/08/briefing/ 2008-4345b1-01-FDA.pdf; Broudy VC. & Lin NL. AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine 2004 ;25:52-60UR - https://www.scopus.com/inward/record.uri?eid=2-s2.0-69849104806&partnerID=40&md5=16444570603467b33d451ed94b961e90",
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language = "English",
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journal = "Journal of Clinical and Diagnostic Research",
issn = "2249-782X",
publisher = "Journal of Clinical and Diagnostic Research",
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Romiplostim: A novel c-Mpl/CD110 receptor ligand for the management of idiopathic thrombocytopenic purpura. / Patel, H.; Patel, N.; Vyas, A.; Patel, M.; Pandey, S.

In: Journal of Clinical and Diagnostic Research, Vol. 3, No. 4, 2009, p. 1690-1696.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Romiplostim: A novel c-Mpl/CD110 receptor ligand for the management of idiopathic thrombocytopenic purpura

AU - Patel, H.

AU - Patel, N.

AU - Vyas, A.

AU - Patel, M.

AU - Pandey, S.

N1 - Cited By :1 Export Date: 10 November 2017 Correspondence Address: Patel, H.223 Raman Block, Udupi, Karnataka 576104, India; email: hiren379@gmail.com Chemicals/CAS: cyclophosphamide, 50-18-0; danazol, 17230-88-5; immunoglobulin, 9007-83-4; rituximab, 174722-31-7; romiplostim, 267639-76-9, 847595-08-8 References: George, J.N., El-Harake, M.A., Raskob, G.E., Chronic idiopathic thrombocytopenic purpura (1994) N Engl J Med, 331 (18), pp. 1207-1211. , Nov 3;; Karpatkin, S., Autoimmune (idiopathic) thrombocytopenic purpura (1997) Lancet, 349 (9064), pp. 1531-1536. , May 24;; Cohen, Y.C., Djulbegovic, B., Shamai-Lubovitz, O., Mozes, B., The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts (2000) Arch Intern Med, 160 (11), pp. 1630-1638. , Jun 12;; George, J.N., Kojouri, K., Perdue, J.J., Vesely, S.K., Management of patients with chronic, refractory idiopathic thrombocytopenic purpura (2000) Semin Hematol, 37 (3), pp. 290-298. , Jul;; George, J.N., Woolf, S.H., Raskob, G.E., Wasser, J.S., Aledort, L.M., Ballem, P.J., Idiopathic thrombocytopenic purpura: A practice guideline developed by explicit methods for the American Society of Hematology (1996) Blood, 88 (1), pp. 3-40. , Jul 1;; Scaradavou, A., Woo, B., Woloski, B.M., Cunningham-Rundles, S., Ettinger, L.J., Aledort, L.M., Intravenous anti-D treatment of immune thrombocytopenic purpura: Experience in 272 patients (1997) Blood, 89 (8), pp. 2689-2700. , Apr 15;; Stasi, R., Provan, D., Management of immune thrombocytopenic purpura in adults (2004) Mayo Clin Proc, 79 (4), pp. 504-522. , Apr;; McMillan, R., Wang, L., Tomer, A., Nichol, J., Pistillo, J., Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP (2004) Blood, 103 (4), pp. 1364-1369. , Oct 23;; Heyns, A.D., Lotter, M.G., Badenhorst, P.N., Kock, F.D., Pieters, H., Herbst, C., Kinetics and sites of destruction of 111Indium-oxine-labeled platelets in idiopathic thrombocytopenic purpura: A quantitative study (1982) Am J Hematol, 12 (2), pp. 167-177; Ballem, P.J., Segal, G.M., Stratton, J.R., Gernsheimer, T., Adamson, J.W., Slichter, S.J., Mechanisms of thrombocytopenia in chronic autoimmune thrombocytopenic purpura. Evidence of both impaired platelet production and increased platelet clearance (1987) J Clin Invest, 80 (1), pp. 33-40. , Jul;; Nichol, J.L., Thrombopoietin levels after chemotherapy and in naturally occurring human diseases (1998) Curr Opin Hematol, 5 (3), pp. 203-208. , May;; Kosugi, S., Kurata, Y., Tomiyama, Y., Circulating thrombopoietin level in chronic immune thrombocytopenic purpura (1996) Br J Haematol, 93 (3), pp. 704-706; Emmons, R.V., Reid, D.M., Cohen, R.L., Human thrombopoietin levels are high when thrombocytopenia is due to megakaryocyte deficiency and low when due to increased platelet destruction (1996) Blood, 87, pp. 4068-4071; Houwerzijl, E.J., Blom, N.R., van der Want, J.J., Esselink, M.T., Koornstra, J.J., Smit, J.W., Ultrastructural study shows morphologic features of apoptosis and para-apoptosis in megakaryocytes from patients with idiopathic thrombocytopenic purpura (2004) Blood, 103 (2), pp. 500-506. , Jan 15;; Kuter, D.J., Bussel, J.B., Lyons, R.M., Pullarkat, V., Gernsheimer, T.B., Senecal, F.M., Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: A double-blind randomized controlled trial (2002) Lancet, 371, pp. 395-403. , Feb 2;; Kuter, D.J., New thrombopoietic growth factors (2007) Blood, 109 (11), pp. 4607-4616. , Jun 1;; Bussel, J.B., Kuter, D.J., George, J.N., Mcmillan, R., Aledort, L.M., Conklin, G.T., AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP (2006) N Engl J Med, 355 (16), pp. 1672-1681. , Oct 19;; Bussel JB, Kutter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood [serial online ] 2008 Nov 3 [cited 2009 Jan 28]: Available from: URL: http://bloodjournal.hematologylibrary.org/ cgi/content/abstract/blood-2008-04-150078v1; Highlights of prescribing information of romiplostim, , http://www.fda.gov/cder/foi/label/2008/125268lbl.pdf, Online, 2008 Aug [cited 2009 Jan 28, 1,2,6,8. Available from: URL; Peetersk, K., Stassen, J.M., Collen, D., Geet, C.V., Freson, K., Emerging treatments for thrombocytopenia: Increasing platelet production (2008) Drug discovery today, 13 (17-18), pp. 798-806. , Sep;; Wang, B., Nichol, J.L., Sullivan, J.T., Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor (2004) Clin Pharmacol Ther, 76 (6), pp. 628-638; Stasi, R., Evangelista, M.L., Amadori, S., Novel thrombopoietic agents: A review of their use in idiopathic thrombocytopenia purpura (2008) Drugs, 68 (7), pp. 901-912; Methia, N., Louache, F., Vainchenker, W., Wendling, F., Oligodeoxynucleotides antisense to the proto-oncogene c-mpl specifically inhibit in vitro megakaryopoiesis (1993) Blood, 82 (5), pp. 1395-1401. , Sep 1;; Lodish, H., Berk, A., Kaiser, C.A., Krieger, M., Scott, M.P., Bretscher, A., (2008) Molecular cell biology, pp. 672-676. , 6th ed. New York: W. H. Freeman and Company;, P; Clinical Pharmacology and biopharmaceutics Reviews of romiplostim. Food and drug Administration, , http://www.fda.gov/cder/foi/nda/2008/125268s000_ClinPharmR.pdf, online, cited 2009 Jan 28];Available from: URL; Kumagi, Y., Fujita, T., Ozaki, M., Sahashi, K., Ohkura, M., Ohtsu, T., Pharmacodynamics and pharmacokinetics of AMJ531, a Thrombopoesis Stimulating peptibody, in Healthy Japanese Subjects: A Randomized, Placebo Controlled study (2007) J Clin Pharmacol, 47, pp. 1489-1497; Newland, A., Caulier, M.T., Kappers, K.M., An open label, unit dose finding study of AMG 531, a novel thrombopoesis stimulating peptibody in patient with ITP (2006) Br J Haematol, 135, pp. 547-553; George J, Bussel JB, Lyons RM, Pullarkat V, Redner R, Terreu D. et al. Self injection of Romiplostim by patients with chronic immune thrombocytopenic Purpura(ITP). Blood [serial online] 2008 Dec [cited 2009 Jan 29]; 112: Available from : URL: http://www.cancercaresouthtexas.com/docs/ SelfInjection%20of%20Romiplostim%20by%20 Patients%20with%20Chronic%20Immune%20Throbocytopeni.pdf; Cines, D.B., Yasothan, U., Kirkpatrick, P., Romiplostim, (2008) Nature Review Drug Discovery, pp. 887-888. , Nov 7; Oncologic drug advisory committee. FDA advisory committee briefing document on romiplostim [Online]. 2008 Mar [cited 2009 Jan 25]; 3,6-9. Available from: URL: http://www.fda.gov/ohrms/dockets/AC/08/briefing/ 2008-4345b1-01-FDA.pdf; Broudy VC. & Lin NL. AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine 2004 ;25:52-60UR - https://www.scopus.com/inward/record.uri?eid=2-s2.0-69849104806&partnerID=40&md5=16444570603467b33d451ed94b961e90

PY - 2009

Y1 - 2009

N2 - Idiopathic Thrombocytopenic Purpura (ITP) is an autoimmune disease characterised predominantly by an antibody mediated destruction of platelets. Few treatment options are available for ITP like corticosteroids, IV immunoglobulins, splenectomy, rituximab, danazole and cyclophosphamide. All these treatment options work by reducing the destruction of platelets by antibody. However recent evidence suggests that production of platelet is reduced in 75% of thrombocytopenic patient with ITP. Romiplostim is a novel thrombopoetin receptor agonist approved by USFDA in August 2008 that increases the platelet count in ITP patients unresponsive to corticosteroids, immunoglobulins or splenectomy. Romiplostim is not currently available in India. The addition of Romiplostim in the treatment options for ITP will surely improve the outcomes in ITP patients.

AB - Idiopathic Thrombocytopenic Purpura (ITP) is an autoimmune disease characterised predominantly by an antibody mediated destruction of platelets. Few treatment options are available for ITP like corticosteroids, IV immunoglobulins, splenectomy, rituximab, danazole and cyclophosphamide. All these treatment options work by reducing the destruction of platelets by antibody. However recent evidence suggests that production of platelet is reduced in 75% of thrombocytopenic patient with ITP. Romiplostim is a novel thrombopoetin receptor agonist approved by USFDA in August 2008 that increases the platelet count in ITP patients unresponsive to corticosteroids, immunoglobulins or splenectomy. Romiplostim is not currently available in India. The addition of Romiplostim in the treatment options for ITP will surely improve the outcomes in ITP patients.

M3 - Article

VL - 3

SP - 1690

EP - 1696

JO - Journal of Clinical and Diagnostic Research

JF - Journal of Clinical and Diagnostic Research

SN - 2249-782X

IS - 4

ER -