Successful anticoagulant therapy for two pregnant PNH patients, and prospects for the eculizumab era

详细信息    查看全文

• 作者：Yasuyoshi Morita (1)
  Jun-ichi Nishimura (2)
  Takahiro Shimada (1)
  Hirokazu Tanaka (1)
  Kentaro Serizawa (1)
  Yasuhiro Taniguchi (1)
  Mitsuhiro Tsuritani (3)
  Yuzuru Kanakura (2)
  Itaru Matsumura (1)
  
• 关键词：Paroxysmal nocturnal hemoglobinuria ; Pregnancy ; Low ; molecular weight heparin ; Eculizumab
• 刊名：International Journal of Hematology
• 出版年：2013
• 出版时间：April 2013
• 年：2013
• 卷：97
• 期：4
• 页码：491-497
• 全文大小：183KB
• 参考文献：1. Parker C, Omine M, Richards S, Nishimura J, Bessler M, Ware R, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106:3699鈥?09. CrossRef
  2. Takeda J, Miyata T, Kawagoe K, Iida Y, Endo Y, Fujita T, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993;73:703鈥?1. CrossRef
  3. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal hemoglobinuria. N Engl J Med. 1995;333:1253鈥?. CrossRef
  4. Shichishima T, Saitoh Y, Noji H, Terasawa T, Maruyama Y. In vivo effects of various therapies on complement-sensitive erythrocytes in paroxysmal nocturnal hemoglobinuria. Int J Hematol. 1996;6:291鈥?02. CrossRef
  5. Issaragrisil S, Piankijagum A, Tang-naitrisorana Y. Corticosteroids therapy in paroxysmal nocturnal hemoglobinuria. Am J Hematol. 1987;25:77鈥?3. CrossRef
  6. Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004;350:552鈥?. CrossRef
  7. Hillmen P, Young NS, Schubert J, Brodsky RA, Soci茅 G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233鈥?3. CrossRef
  8. Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111:1840鈥?. CrossRef
  9. Kanakura Y, Ohyashiki K, Schishima T, Okamoto S, Ando K, Ninomiya H, et al. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011;93:36鈥?6. CrossRef
  10. Socie G, Mary JY, de Gramont A, Rio B, Leporrier M, Rose C, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996;31:573鈥?. CrossRef
  11. Hillmen P, Muus P, D眉hrsen U, Risitano AM, Schubert J, Luzzatto L, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110:4123鈥?. CrossRef
  12. Nishimura J, Kanakura Y, Ware RE, Shichishima T, Nakakuma H, Ninomiya H, et al. Clinical course and flow cytometric analysis of paroxysmal nocturnal hemoglobinuria in the United States and Japan. Medicine. 2004;83:193鈥?07. CrossRef
  13. Bais J, Pel M, von dem Borne A, van der Lelie H. Pregnancy and paroxysmal nocturnal hemoglobinuria. Eur J Obstet Gynecol Reprod Biol. 1994;53:211鈥?. CrossRef
  14. Tichelli A, Socie G, Marsh J, Barge R, Frickhofen N, McCann S, et al. Outcome of pregnancy and disease course among women with aplastic anemia treated with immunosuppression. Ann Intern Med. 2002;136:164鈥?2.
  15. James AH, Tapson VF, Goldhaber SZ. Thrombosis during pregnancy and the postpartum period. Am J Obstet Gynecol. 2005;193:216鈥?. CrossRef
  16. Ray JG, Burows RF, Ginsberg JS, Burrows EA. Paroxysmal nocturnal hemoglobinuria and the risk of venous thrombosis: review and recommendations for management of the pregnant and nonpregnant patient. Haemostasis. 2000;30:103鈥?7.
  17. Hall C, Richards S, Hillmen P. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003;102:3587鈥?1. CrossRef
  18. Ray JG, Chan WS. Deep vein thrombosis during pregnancy and the puerperium: a meta-analysis of the period of risk and the leg of presentation. Obstet Gynecol Surv. 1999;54:265鈥?1. CrossRef
  19. Moyo VM, Mukhina GL, Garrett ES, Brodsky RA. Natural history of paroxysmal nocturnal haemoglobinuria using modern diagnostic assays. Br J Haematol. 2004;126:133鈥?. CrossRef
  20. Danilov AV, Brodsky RA, Craigo S, Smith H, Miller KB. Managing a pregnant patient with paroxysmal nocturnal hemoglobinuria in the era of eculizumab. Leuk Res. 2010;34:566鈥?1. CrossRef
  21. Greer IA, Nelson-Piercy C. Low-molecular-weight heparins for thromboprophylaxis and treatment of venous thromboembolism in pregnancy: a systematic review of safety and efficacy. Blood. 2005;106:401鈥?. CrossRef
  22. Meyers G, Parker CJ. Management issues in paroxysmal nocturnal hemoglobinuria. Int J Hematol. 2003;77:125鈥?2. CrossRef
  23. Bj酶rge L, Ernst P, Haram KO. Paroxysmal nocturnal hemoglobinuria in pregnancy. Acta Obstet Gynecol Scand. 2003;82:1067鈥?1. CrossRef
  24. Allen TK, George RB, Olufolabi AJ, James AH, Muir HA, Habib AS. The management of Cesarean delivery in a parturient with paroxysmal nocturnal hemoglobinuria complicated by severe preeclampsia. Can J Anaesth. 2007;54:646鈥?1. CrossRef
  25. Solal-C茅ligny P, Tertian G, Fernandez H, Pons JC, Lambert T, Najean Y, et al. Pregnancy and paroxysmal nocturnal hemoglobinuria. Arch Intern Med. 1988;148:593鈥?. CrossRef
  26. de Guibert S, Peffault de Latour R, Varoqueaux N, Labussi猫re H, Rio B, Jaulmes D, et al. Paroxysmal nocturnal hemoglobinuria and pregnancy before the eculizumab era: the French experience. Haematologica. 2011;96:1276鈥?3.
  27. Melo A, Gorgal-Carvalho R, Amaral J, Marques MC, Andrade J, Guimar茫es JT, et al. Clinical management of paroxysmal nocturnal haemoglobinuria in pregnancy: three case reports. Blood Transfus. 2011;9:99鈥?03.
  28. Greene MF, Frigoletto FD Jr, Claster S, Rosenthal D. Pregnancy and paroxysmal nocturnal hemoglobinuria: report of a case and review of the literature. Obstet Gynecol Surv. 1983;38:591鈥?. CrossRef
  29. Shibasaki T, Matsuda H, Furuya K. Haptoglobin therapy during pregnancy for paroxysmal nocturnal hemoglobinuria with renal failure. Int J Gynaecol Obstet. 2007;98:267鈥?. CrossRef
  30. Chen ML, Yu CH, Chang FM, Kuo PL. Paroxysmal nocturnal hemoglobinuria superimposed with preeclampsia. Taiwan J Obstet Gynecol. 2006;45:276鈥?. CrossRef
  31. Fieni S, Bonfanti L, Gramellini D, Benassi L, Delsignore R. Clinical management of paroxysmal nocturnal hemoglobinuria in pregnancy: a case report and updated review. Obstet Gynecol Surv. 2006;61:593鈥?01. CrossRef
  32. Paech MJ, Pavy TJ. Management of a parturient with paroxysmal nocturnal haemoglobinuria. Int J Obstet Anesth. 2004;13:188鈥?1. CrossRef
  33. Kjaer K, Comerford M, Gadalla F. General anesthesia for cesarean delivery in a patient with paroxysmal nocturnal hemoglobinuria and thrombocytopenia. Anesth Analg. 2004;98:1471鈥?. CrossRef
  34. Higgins SP, McMahon LP, Brennecke SP. Paroxysmal nocturnal haemoglobinuria in pregnancy鈥攏ot to be confused with pre-eclampsia or HELLP syndrome. Case report and literature review. J Obstet Gynaecol. 2004;24:83鈥?. CrossRef
  35. Stocche RM, Garcia LV, Klamt JG. Labor analgesia in a patient with paroxysmal nocturnal hemoglobinuria with thrombocytopenia. Reg Anesth Pain Med. 2001;26:79鈥?2.
  36. Svigos JM, Norman J. Paroxysmal nocturnal haemoglobinuria and pregnancy. Aust N Z J Obstet Gynaecol. 1994;34:104鈥?. CrossRef
  37. Imai A, Takagi H, Kawabata I, Sumi H, Shiraki S, Tamaya T. Successful pregnancy in a patient with paroxysmal nocturnal hemoglobinuria: case report. Arch Gynecol Obstet. 1989;246:121鈥?. CrossRef
  38. Mori H, Okada S, Takizawa Y, Hagiwara S, Niikura H, Terada H. Pregnancy in paroxysmal nocturnal hemoglobinuria鈥搑eport of two cases. Rinsho Ketsueki. 1987;28:1645鈥?0.
  39. Beresford CH, Gudex DJ, Symmans WA. Paroxysmal nocturnal haemoglobinuria and pregnancy. Lancet. 1986;2:1396鈥?. CrossRef
  40. Jacobs P, Wood L. Paroxysmal nocturnal haemoglobinuria and pregnancy. Lancet. 1986;2:1099. CrossRef
  41. Tomaru T, Nakai K, Takeda S, Kagawa T, Ozawa K. Anesthetic management of caesarean section in a patient with paroxysmal nocturnal hemoglobinuria. Masui. 1986;35:807鈥?1.
  42. Colvin BT. Thrombocytopenia. Clin Haematol. 1985;14:661鈥?1.
  43. Hurd WW, Miodovnik M, Stys SJ. Pregnancy associated with paroxysmal nocturnal hemoglobinuria. Obstet Gynecol. 1982;60:742鈥?.
  44. Wozniak AJ, Kitchens CS. Prospective hemostatic studies in a patient having paroxysmal nocturnal hemoglobinuria, pregnancy, and cerebral venous thrombosis. Am J Obstet Gynecol. 1982;142:591鈥?.
  45. Kawamura S, Shiosaka T, Saito S, Ikeda K, Matsumoto I, Fujita S, et al. A case of paroxysmal nocturnal hemoglobinuria; successful gestation and labor. Rinsho Ketsueki. 1980;21:1226鈥?1.
  46. Spencer JA. Paroxysmal nocturnal haemoglobinuria in pregnancy: case report. Br J Obstet Gynaecol. 1980;87:246鈥?. CrossRef
  47. Frakes JT, Burmeister RE, Giliberti JJ. Pregnancy in a patient with paroxysmal nocturnal hemoglobinuria. Obstet Gynecol. 1976;47:22S鈥?S.
  48. Payne PR. Paroxysmal nocturnal haemoglobinuria in pregnancy. Proc R Soc Med. 1968;61:679.
  49. R枚th A, Hock C, Konik A, Christoph S, D眉hrsen U. Chronic treatment of paroxysmal nocturnal hemoglobinuria patients with eculizumab: safety, efficacy, and unexpected laboratory phenomena. Int J Hematol. 2011;93:704鈥?4. CrossRef
  50. Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, et al. The management of pregnancy in paroxysmal nocturnal haemoglobinuria on long term eculizumab. Br J Haematol. 2010;149:446鈥?0. CrossRef
  51. Figueroa JE, Densen P. Infectious diseases associated with complement deficiencies. Clin Microbiol Rev. 1991;4:359鈥?5.
  52. Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L. Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol. 2007;25:1256鈥?4. CrossRef
  53. van Bijnen ST, van Rijn RS, Koljenovic S, Te Boekhorst P, de Witte T, Muus P. Possible high risk of thrombotic events in patients with paroxysmal nocturnal haemoglobinuria after discontinuation of eculizumab. Br J Haematol. 2012;157:762鈥?. CrossRef
  54. Marasca R, Coluccio V, Santachiara R, Leonardi G, Torelli G, Notaro R, et al. Pregnancy in PNH: another eculizumab baby. Br J Haematol. 2010;150:707鈥?. CrossRef
  
• 作者单位：Yasuyoshi Morita (1)
  Jun-ichi Nishimura (2)
  Takahiro Shimada (1)
  Hirokazu Tanaka (1)
  Kentaro Serizawa (1)
  Yasuhiro Taniguchi (1)
  Mitsuhiro Tsuritani (3)
  Yuzuru Kanakura (2)
  Itaru Matsumura (1)
  
  1. Division of Hematology and Rheumatology, Department of Internal Medicine, Kinki University Faculty of Medicine, Osaka-Sayama, Osaka, 589-8511, Japan
  2. Department of Hematology and Oncology, Osaka University, Graduate School of Medicine, Suita, Osaka, Japan
  3. Department of Obstetrics and Gynecology, Kinki University Faculty of Medicine, Osaka-Sayama, Osaka, Japan
  

文摘

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired stem cell disorder characterized by intravascular hemolysis and thrombosis. The most serious complication is thrombosis, the risk of which is augmented by the hyper-coagulable state that occurs during pregnancy; despite this risk, however, young female PNH patients often desire to have a baby. We recently experienced two successful deliveries in PNH patients, who were treated with anticoagulant therapy during their pregnancies. Meanwhile, given the potential benefit of eculizumab (Soliris), a humanized monoclonal antibody against C5, in reducing thrombosis and hemolysis, it represents a promising therapeutic option for the treatment of pregnant PNH patients in combination with, or in replacement of, anticoagulant therapy.