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1. Full Text A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A
by Peyvandi, Flora and Mannucci, Pier M and Garagiola, Isabella and El-Beshlawy, Amal and Elalfy, Mohsen and Ramanan, Vijay and Eshghi, Peyman and Hanagavadi, Suresh and Varadarajan, Ramabadran and Karimi, Mehran and Manglani, Mamta V and Ross, Cecil and Young, Guy and Seth, Tulika and Apte, Shashikant and Nayak, Dinesh M and Santagostino, Elena and Mancuso, Maria Elisa and Sandoval Gonzalez, Adriana C and Mahlangu, Johnny N and Bonanad Boix, Santiago and Cerqueira, Monica and Ewing, Nadia P and Male, Christoph and Owaidah, Tarek and Soto Arellano, Veronica and Kobrinsky, Nathan L and Majumdar, Suvankar and Perez Garrido, Rosario and Sachdeva, Anupam and Simpson, Mindy and Thomas, Mathew and Zanon, Ezio and Antmen, Bulent and Kavakli, Kaan and Manco-Johnson, Marilyn J and Martinez, Monica and Marzouka, Esperanza and Mazzucconi, Maria G and Neme, Daniela and Palomo Bravo, Angeles and Paredes Aguilera, Rogelio and Prezotti, Alessandra and Schmitt, Klaus and Wicklund, Brian M and Zulfikar, Bulent and Rosendaal, Frits R
The New England Journal of Medicine, ISSN 0028-4793, 05/2016, Volume 374, Issue 21, pp. 2054 - 2064
In a randomized, multicenter trial involving boys with severe hemophilia A, the incidence of neutralizing antibodies to factor VIII was 87% higher with... 
UNITED-STATES | MORTALITY | CONCENTRATE | MEDICINE, GENERAL & INTERNAL | PLASMA | PRODUCTS | RECOMBINANT | INHIBITOR DEVELOPMENT | FVIII | PREVIOUSLY UNTREATED PATIENTS | CHILDREN | Factor VIII - immunology | Humans | Isoantibodies - analysis | Middle Aged | Child, Preschool | Infant | Male | Injections, Subcutaneous - adverse effects | von Willebrand Factor - therapeutic use | Incidence | Dose-Response Relationship, Drug | Young Adult | Hemorrhage - etiology | Factor VIII - antagonists & inhibitors | Adult | Drug Therapy, Combination | Child | Factor VIII - therapeutic use | Hemophilia A - immunology | Proportional Hazards Models | Hemophilia A - complications | Adolescent | Aged | Hemophilia A - drug therapy | Antibodies, Neutralizing - blood | Viral antibodies | Care and treatment | Usage | Hemophilia | Antibodies | Clinical trials | Dosage and administration | Coagulation factors | Immunoglobulins | Inhibitor drugs | Von Willebrand factor | Alloantibodies | FDA approval | Regression analysis | Drug therapy | Health risk assessment | Patients
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2. Full Text Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
by Mahlangu, Johnny and Powell, Jerry S and Ragni, Margaret V and Chowdary, Pratima and Josephson, Neil C and Pabinger, Ingrid and Hanabusa, Hideji and Gupta, Naresh and Kulkarni, Roshni and Fogarty, Patrick and Perry, David and Shapiro, Amy and Pasi, K. John and Apte, Shashikant and Nestorov, Ivan and Jiang, Haiyan and Li, Shuanglian and Neelakantan, Srividya and Cristiano, Lynda M and Goyal, Jaya and Sommer, Jurg M and Dumont, Jennifer A and Dodd, Nigel and Nugent, Karen and Vigliani, Gloria and Luk, Alvin and Brennan, Aoife and Pierce, Glenn F and A-LONG Investigators
Blood, ISSN 0006-4971, 01/2014, Volume 123, Issue 3, pp. 317 - 325
This phase 3 pivotal study evaluated the safety, efficacy, and pharmacokinetics of a recombinant FVIII Fc fusion protein (rFVIIIFc) for prophylaxis, treatment... 
TRIAL | DESIGN | F8 | THERAPY | ON-DEMAND | SAFETY | PROLONGED ACTIVITY | INHIBITOR DEVELOPMENT | HEMATOLOGY | VON-WILLEBRAND-FACTOR | Drug Administration Schedule | Factor VIII - pharmacokinetics | Humans | Middle Aged | Recombinant Fusion Proteins - therapeutic use | Male | Treatment Outcome | Hemorrhage - prevention & control | Young Adult | Hemorrhage - drug therapy | Time Factors | Recombinant Fusion Proteins - pharmacokinetics | Adolescent | Adult | Aged | Immunoglobulin Fc Fragments - therapeutic use | Hemophilia A - drug therapy | Child | Factor VIII - therapeutic use | Plenary Paper
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3. Full Text Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A
by Shima, Midori and Hanabusa, Hideji and Taki, Masashi and Matsushita, Tadashi and Sato, Tetsuji and Fukutake, Katsuyuki and Fukazawa, Naoki and Yoneyama, Koichiro and Yoshida, Hiroki and Nogami, Keiji
The New England Journal of Medicine, ISSN 0028-4793, 05/2016, Volume 374, Issue 21, pp. 2044 - 2053
Emicizumab is a humanized bispecific antibody that mimics the cofactor function of factor VIII. In a dose-escalation study in Japanese persons with hemophilia... 
PROPHYLAXIS | DEFINITIONS | MEDICINE, GENERAL & INTERNAL | FACTORS IXA | QUALITY-OF-LIFE | INHIBITORS | MODEL | IMMUNE TOLERANCE | HEMOSTASIS | JOINT BLEEDS | ACE910 | Antibodies, Monoclonal, Humanized - therapeutic use | Factor IX - analysis | Antibodies, Bispecific - pharmacology | Hemophilia A - immunology | Humans | Middle Aged | Male | Hemorrhage - prevention & control | Injections, Subcutaneous - adverse effects | Hemophilia A - complications | Dose-Response Relationship, Drug | Antibodies, Bispecific - therapeutic use | Factor X - analysis | Antibodies, Monoclonal, Humanized - blood | Antibodies, Monoclonal, Humanized - pharmacology | Hemorrhage - etiology | Adolescent | Factor VIII - antagonists & inhibitors | Adult | Antibodies, Bispecific - blood | Drug Therapy, Combination | Hemophilia A - drug therapy | Child | Factor VIII - therapeutic use | Immunoglobulins | Inhibitor drugs | Intravenous administration | Body weight | Hemorrhage | Patients | Bleeding | Clotting | Mimicry | Thromboplastin | Coagulation factors | Hemophilia | Alloantibodies | Children | Drug therapy | Bispecific antibodies
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4. Full Text Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia
by Manco-Johnson, Michael L and Manco-Johnson, Marilyn J and Abshire, Thomas C and Shapiro, Amy D and Riske, Brenda and Hacker, Michele R and Kilcoyne, Ray and Ingram, J. David and Funk, Sharon and Jacobson, Linda and Valentino, Leonard A and Hoots, W. Keith and Buchanan, George R and DiMichele, Donna and Recht, Michael and Brown, Deborah and Leissinger, Cindy and Bleak, Shirley and Cohen, Alan and Mathew, Prasad and Matsunaga, Alison and Medeiros, Desiree and Nugent, Diane and Thomas, Gregory A and Thompson, Alexis A and McRedmond, Kevin and Soucie, J. Michael and Austin, Harlan and Evatt, Bruce L
The New England Journal of Medicine, ISSN 0028-4793, 08/2007, Volume 357, Issue 6, pp. 535 - 544
This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with... 
MEDICINE, GENERAL & INTERNAL | THERAPY | ARTHROPATHY | EXPERIENCE | SCALE | LIFE EXPECTANCY | CARE | CHILDREN | Drug Administration Schedule | Follow-Up Studies | Hemarthrosis - complications | Humans | Child, Preschool | Infant | Male | Treatment Outcome | Hemorrhage - prevention & control | Hemophilia A - complications | Joint Diseases - prevention & control | Joint Diseases - etiology | Hemarthrosis - drug therapy | Factor VIII - administration & dosage | Infusions, Intravenous | Hemarthrosis - prevention & control | Hemophilia A - drug therapy | Child | Prevention | Complications and side effects | Care and treatment | Joint diseases | Hemophilia | Medical research | Hemorrhage
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5. Full Text Interferon-free therapy with direct acting antivirals for HCV/HIV-1 co-infected Japanese patients with inherited bleeding disorders
by Uemura, Haruka and Tsukada, Kunihisa and Mizushima, Daisuke and Aoki, Takahiro and Watanabe, Koji and Kinai, Ei and Teruya, Katsuji and Gatanaga, Hiroyuki and Kikuchi, Yoshimi and Sugiyama, Masaya and Mizokami, Masashi and Oka, Shinichi
PLoS ONE, ISSN 1932-6203, 10/2017, Volume 12, Issue 10, p. e0186255
Introduction Almost 30 years ago, about 30% of Japanese hemophiliacs became infected with HIV-1 and hepatitis C virus (HCV) after receiving contaminated blood... 
HIV-1 | HEPATOCELLULAR-CARCINOMA | SOFOSBUVIR | MULTIDISCIPLINARY SCIENCES | CHRONIC HEPATITIS-C | RISK | COINFECTION | HCV | HUMAN-IMMUNODEFICIENCY-VIRUS | LEDIPASVIR | ALPHA-FETOPROTEIN | HIV-1 - pathogenicity | Hemophilia A - virology | Coinfection - drug therapy | Humans | Middle Aged | Hepatitis C, Chronic - virology | Imidazoles - administration & dosage | Hepacivirus - pathogenicity | Male | Interferons - therapeutic use | Hepatitis C, Chronic - complications | Coinfection - complications | Drug Therapy, Combination - adverse effects | Benzimidazoles - administration & dosage | Adult | Female | Fluorenes - administration & dosage | Hepacivirus - drug effects | Liver Cirrhosis - drug therapy | HIV-1 - drug effects | Liver Cirrhosis - complications | HIV Infections - virology | Hepatitis C, Chronic - drug therapy | Antiviral Agents - administration & dosage | Coinfection - virology | Hemophilia A - complications | Liver Cirrhosis - virology | Sofosbuvir - administration & dosage | Antiviral Agents - adverse effects | HIV Infections - complications | HIV Infections - drug therapy | Hemophilia A - drug therapy | RNA, Viral - drug effects | Viral Load - drug effects | Antiviral agents | Complications and side effects | Dosage and administration | Research | Hepatitis C | Drug therapy | HIV infection | Therapy | Disease | Disorders | Viruses | Infections | Genomes | Drug resistance | Hemorrhage | Bleeding | Ribavirin | Arteritis | Hepatitis | Liver cancer | Acquired immune deficiency syndrome--AIDS | Antiretroviral agents | Hepatology | Human immunodeficiency virus--HIV | Hemophilia | Product safety | Genotypes | Blood products | Glycosylation | Patients | Antiretroviral therapy | Medicine | Studies | Cirrhosis | Interferon | Hepatitis C virus | Prolongation | Acquired immune deficiency syndrome | AIDS | HIV | Human immunodeficiency virus
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6. Full Text Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors
by Mahlangu, Johnny and Oldenburg, Johannes and Paz-Priel, Ido and Negrier, Claude and Niggli, Markus and Mancuso, M. Elisa and Schmitt, Christophe and Jiménez-Yuste, Victor and Kempton, Christine and Dhalluin, Christophe and Callaghan, Michael U and Bujan, Willem and Shima, Midori and Adamkewicz, Joanne I and Asikanius, Elina and Levy, Gallia G and Kruse-Jarres, Rebecca
The New England Journal of Medicine, ISSN 0028-4793, 08/2018, Volume 379, Issue 9, pp. 811 - 822
Emicizumab binds to activated factor IX and factor X, reproducing the bridging function of the missing factor VIII. In a trial of emicizumab prophylaxis,... 
MEDICINE, GENERAL & INTERNAL | BISPECIFIC ANTIBODY | REPLACEMENT THERAPY | GENE-THERAPY | QUALITY-OF-LIFE | MODEL | BURDEN | Antibodies, Monoclonal, Humanized - adverse effects | Antibodies, Monoclonal, Humanized - therapeutic use | Drug Administration Schedule | Hemorrhage - epidemiology | Humans | Middle Aged | Antibodies, Bispecific - pharmacokinetics | Male | Hemorrhage - prevention & control | Injections, Subcutaneous - adverse effects | Young Adult | Antibodies, Bispecific - therapeutic use | Antibodies, Bispecific - adverse effects | Antibodies, Monoclonal, Humanized - pharmacokinetics | Adolescent | Quality of Life | Adult | Aged | Blood Coagulation Factor Inhibitors | Hemophilia A - drug therapy | Factor VIII - therapeutic use | Treatment outcome | Drug therapy | Analysis | Hemophilia | Coagulation factors | Fees & charges | Thrombotic microangiopathy | Hemostasis | Body weight | Prophylaxis | Monoclonal antibodies | Bleeding | Pharmaceuticals
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7. Full Text Emicizumab Prophylaxis in Hemophilia A with Inhibitors
by Oldenburg, Johannes and Mahlangu, Johnny N and Kim, Benjamin and Schmitt, Christophe and Callaghan, Michael U and Young, Guy and Santagostino, Elena and Kruse-Jarres, Rebecca and Negrier, Claude and Kessler, Craig and Valente, Nancy and Asikanius, Elina and Levy, Gallia G and Windyga, Jerzy and Shima, Midori
The New England Journal of Medicine, ISSN 0028-4793, 08/2017, Volume 377, Issue 9, pp. 809 - 818
Inhibitors develop in many patients with hemophilia who receive recombinant factor VIII. Prophylaxis with emicizumab, an antibody that functionally replaces... 
EQ-5D | MEDICINE, GENERAL & INTERNAL | BISPECIFIC ANTIBODY | QUALITY-OF-LIFE | UTILITY | Recombinant Proteins - therapeutic use | Factor VIII - immunology | Thrombosis - chemically induced | Humans | Middle Aged | Antibodies, Bispecific - pharmacokinetics | Male | Hemorrhage - prevention & control | Factor VIIa - therapeutic use | Isoantibodies - blood | Young Adult | Antibodies, Monoclonal, Humanized - pharmacokinetics | Injections, Subcutaneous | Adult | Drug Therapy, Combination | Child | Factor VIII - therapeutic use | Antibodies, Monoclonal, Humanized - adverse effects | Antibodies, Monoclonal, Humanized - therapeutic use | Hemophilia A - immunology | Antibodies, Bispecific - therapeutic use | Antibodies, Bispecific - adverse effects | Blood Coagulation Factors - adverse effects | Adolescent | Blood Coagulation Factors - therapeutic use | Aged | Hemophilia A - drug therapy | Care and treatment | Hemorrhage | Analysis | Hemophilia | Thrombotic microangiopathy | Hematology | Prophylaxis | Antibodies | Recovery of function | Thrombosis | Bleeding | Medicine | Coagulation factors | Prothrombin | Drug dosages
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8. Full Text Rituximab in the treatment of autoimmune haematological disorders
by Garvey, Bernadette
British Journal of Haematology, ISSN 0007-1048, 04/2008, Volume 141, Issue 2, pp. 149 - 169
Summary Current treatment regimens for haematological autoimmune diseases are relatively non-selective and are often associated with considerable toxicity.... 
idiopathic thrombocytopenic purpura | thrombotic thrombocytopenic purpura | acquired haemophilia | cold agglutinin disease | autoimmune haemolytic anaemia | rituximab | Rituximab | Autoimmune haemolytic anaemia | Cold agglutinin disease | Idiopathic thrombocytopenic purpura | Thrombotic thrombocytopenic purpura | Acquired haemophilia | HEMOLYTIC-UREMIC SYNDROME | FACTOR-VIII INHIBITORS | B-CELL DEPLETION | ANTI-CD20 MONOCLONAL-ANTIBODY | PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY | CHRONIC LYMPHOCYTIC-LEUKEMIA | NON-HODGKINS-LYMPHOMA | OF-THE-LITERATURE | HEMATOLOGY | Antigens, CD20 - immunology | Antibodies, Monoclonal, Murine-Derived | Humans | Middle Aged | Anemia, Hemolytic, Autoimmune - drug therapy | Hematologic Diseases - drug therapy | Antibodies, Monoclonal - therapeutic use | Purpura, Thrombocytopenic, Idiopathic - drug therapy | Aged, 80 and over | Purpura, Thrombotic Thrombocytopenic - drug therapy | Adult | Aged | Hemophilia A - drug therapy | Autoimmune Diseases - drug therapy | Drug therapy | Hemophilia | Index Medicus
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9. Full Text 2017 Clinical trials update: Innovations in hemophilia therapy
by Hartmann, Jan and Croteau, Stacy E
American Journal of Hematology, ISSN 0361-8609, 12/2016, Volume 91, Issue 12, pp. 1252 - 1260
A surge in therapeutic clinical trials over recent years is paving the way for transformative treatment options for patients with hemophilia. The introduction... 
HUMAN-CL RHFVIII | EXTENDED HALF-LIFE | FC FUSION PROTEIN | FACTOR-IX | RECOMBINANT FACTOR-VIII | PLATELET GENE-THERAPY | COAGULATION-FACTOR VIII | HEMATOLOGY | FACTOR PATHWAY INHIBITOR | ON-DEMAND TREATMENT | IMMUNE TOLERANCE INDUCTION | Blood Coagulation - drug effects | Factor IX - therapeutic use | Humans | Recombinant Fusion Proteins - therapeutic use | Half-Life | Clinical Trials as Topic | Hemophilia A - drug therapy | Factor VIII - therapeutic use | Genetic Therapy - methods | Hemophilia B - drug therapy | Proteins | Viral antibodies | Exercise | Blood coagulation factor VIII | Hemophilia | Clinical trials | Antibodies | Gene therapy | Patient compliance
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10. Full Text International recommendations on the diagnosis and treatment of patients with acquired hemophilia A
by Huth-Kuhne, Angela and Baudo, Francesco and Collins, Peter and Ingerslev, Jorgen and Kessler, Craig M and Levesque, Herve and Castellano, Maria Eva Mingot and Shima, Midori and St-Louis, Jean
Haematologica, ISSN 0390-6078, 04/2009, Volume 94, Issue 4, pp. 566 - 575
1 SRH Kurpfalzkrankenhaus Heidelberg gGmbH and Hemophilia Center, Heidelberg, Germany 2 Thrombosis and Hemostasis Unit, Niguarda Hospital, Milan, Italy 3... 
Acquired hemophilia | Recommendations | Inhibitors | Treatment | Bleeding | RECOMBINANT FACTOR-VIIA | inhibitors | treatment | INTRAVENOUS GAMMA-GLOBULIN | bleeding | acquired hemophilia | FACTOR-VIII INHIBITORS | CENTER DOCTORS ORGANIZATION | recommendations | FACTOR-IX INHIBITORS | IMMUNOSUPPRESSIVE THERAPY | PROTHROMBIN COMPLEX CONCENTRATE | PORCINE FACTOR-VIII | OF-THE-LITERATURE | HEMATOLOGY | BONN-MALMO PROTOCOL | Hemorrhage - drug therapy | Partial Thromboplastin Time | Hemophilia A - etiology | Humans | Hemorrhage - prevention & control | International Cooperation | Drug Therapy, Combination | Hemophilia A - diagnosis | Hemophilia A - drug therapy | Germany | Decision Making and Problem Solving
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11. Full Text An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
by Sehgal, Alfica and Barros, Scott and Ivanciu, Lacramioara and Cooley, Brian and Qin, June and Racie, Tim and Hettinger, Julia and Carioto, Mary and Jiang, Yongfeng and Brodsky, Josh and Prabhala, Harsha and Zhang, Xuemei and Attarwala, Husain and Hutabarat, Renta and Foster, Don and Milstein, Stuart and Charisse, Klaus and Kuchimanchi, Satya and Maier, Martin A and Nechev, Lubo and Kandasamy, Pachamuthu and Kel'In, Alexander V and Nair, Jayaprakash K and Rajeev, Kallanthottathil G and Manoharan, Muthiah and Meyers, Rachel and Sorensen, Benny and Simon, Amy R and Dargaud, Yesim and Negrier, Claude and Camire, Rodney M and Akinc, Akin
Nature Medicine, ISSN 1078-8956, 05/2015, Volume 21, Issue 5, pp. 492 - 497
Hemophilia A and B are inherited bleeding disorders characterized by deficiencies in procoagulant factor VIII (FVIII) or factor IX (FIX), respectively. There... 
MEDICINE, RESEARCH & EXPERIMENTAL | BLEEDING MODEL | ACTIVATED PROTEIN-C | CLINICAL EXPRESSION | BIOCHEMISTRY & MOLECULAR BIOLOGY | FACTOR-VIII | IN-VIVO HEMOSTASIS | DEFICIENCY | A MICE | CELL BIOLOGY | THROMBIN GENERATION | FACTOR-V-LEIDEN | VENOUS THROMBOEMBOLISM | Hemostasis - drug effects | Factor IX - chemistry | Humans | Factor VIII - chemistry | Male | Antithrombins - chemistry | Dose-Response Relationship, Drug | Homozygote | Animals | Blood Coagulation - drug effects | RNA Interference | Female | Hemophilia A - genetics | Mice | Mutation | Hemophilia A - drug therapy | Care and treatment | Antithrombins | RNA | Hemostasis | Hemophilia |