Celebrating 60 Years of Caring NHF Nursing Luncheon November 14, 2008 Jim Munn, RN, MS
Hemophilia: Early Observations - Second Century For it was taught: If she circumcised her first child and he died [as a result of bleeding from the operation] and a second one died [similarly], she must not circumcise her third child. R. Judah, the Patriarch, redactor of the Mishnah Rosner F. Medicine in the Bible and the Talmud, revised ed, New York: Yeshiva University Press, Ktav Publishing House, 1995.
Early Observations Tenth Century Islamic Surgeon Abul Qasim Al-Zahravi (Albucasis) 936 -1013 CE – Considered greatest surgeon of Middle Ages – Referred to a condition interpreted by scholars as hemophilia in his encyclopedic work on medicine and surgery, al-Tasrif Al-Tasrif Uman ‘Ajiza An Al-Taalif.
Early Observations Twelfth Century Physician and Talmudist Maimonides states in the Mishneh Torah 1 “If a woman had her first son circumcised and he died as a result of the circumcision, which enfeebled his strength, and she similarly had her second [son] circumcised and he died as a result of the circumcision - whether [the latter child] was from her first husband or her second husband - the third son may not be circumcised at the proper time [on the eighth day of life]…” 2 1. Rosner F. “Moses Maimonides. ” Ann Intern Med. 1965; 372: 1135 -1204. 2. Mishneh Torah, Hilkhot Milah. 1: 18.
Early Observations 1803: John Conrad Otto n Provided first accurate account of hemophilia in the modern medical literature n His investigation of “bleeders” was published in a New York journal under the title, “An Account of an Hemorrhagic Disposition Existing in Certain Families” n Traced to woman who settled in Plymouth, New Hampshire in 1720 Otto JC. The Medical Repository. 1803; Vol VI (No 1): 1 -4.
Early Observations 1820: Nasse of Bonn – German physician 1778 -1851 – Formulated observations on inheritance of hemophilia – “Nasse’s Law” n Transmitted by unaffected females to their sons Nasse CF. Arch Med Erfahr. 1(1820): 385.
Early Observations n n 1828: The word “hemophilia” first appears in a description of inherited bleeding disorders by Physician Frederick Hopff at the University of Zurich 1840: First recorded case of hemophilia treatment by transfusion written by Samuel Lane in The Lancet 1 n 1893: First documentation of abnormal prolongation of coagulation in capillary tube in hemophilics 2 1. Farr AD. J Royal Soc Med. April 1981; 74(4): 301 -305. 2. Wright AE. Br Med J. 1893; 2: 223 -225.
Early Observations: Other Highlights of the Early 1900 s n n n 1911: Publication of “monumental review of pedigrees of families with bleeding disorders” 1 1920 -1930: Hemophilia treatments published; plasma for transfusions introduced 1926: Erik von. Willebrand describes bleeding disorder affecting both sexes 1937: IV administration of redissolved plasma precipitate shown to shorten blood clotting time 2 1937: First permanent blood bank established in US 1. Ingram GIC. J Clin Pathol. 1976; 6: 3. 2. Brinkhous KM. A Short History of Hemophilia. Handbook of Hemophilia. New York: American Elsevier, 1975.
1940 s Early to Mid-1940 s: Medical progress accompanies war – Charles Drew sets standard for collecting, storing and transporting plasma under “battlefield conditions” – Edwin Cohn, Harvard biochemist, first to use fractionation process to produce albumin in 19441 – Albumin usefulness spurs research in blood component therapy by “clotters” – 1946 -1947: Hemophilia defect in plasma component 2 1. Latham A Jr. Vox Sang. 1986; 51(3): 249 -52. 2. Brinkhous KM. Proc Soc Exper Biol and Med. Oct. 1947; 66: 117 -120.
1940 s 1948: A golden decade for coagulation research begins n Hemophilia Foundation established 6/15/48 n Hemophilic dog colony established: Chapel Hill, North Carolina ©UNC Center for Thrombosis and Hemostasis Smith N. A History of the National Hemophilia Foundation. New York: National Hemophilia Foundation, 1984.
1950 s n n First recommendation for home infusion 1959: First hemophilia treatment center in Rochester, NY Photos of Mary Gooley (above) courtesy of the Mary M. Gooley Hemophilia Center, Rochester, NY Smith N. A History of the National Hemophilia Foundation. New York: National Hemophilia Foundation, 1984.
1950 s Biggs R, et al. Br Med J. Dec. 27, 1952; 1378. With permission from BMJ Publishing Group.
1950 s 1952: Evolution of the definition of hemophilia: – A blood clotting disorder affecting males with two possible major protein deficiencies: • • FVIII - Hemophilia A FIX - Hemophilia B – Thromboplastin generation test for measuring FVIII developed – Coagulation cascade discovered Langdell RD, et al. J Lab Clin Med. 1953; 41: 637 -647.
1950 s 1958: First use of prophylaxis in Hemophilia A conducted in Sweden by Prof. Inga Marie Nilsson
1960 s n n n 1960 s: NIH grants create research climate 1961: Hyland Laboratories begins work on FVIII concentrate 1966: Peanut flour touted as treatment for hemophilia – Nature 1 1. Nature. February 13, 1969. Vol. 185, No. 4711, pp. 469 -470.
1960 s 1965: Discovery of Cryoprecipitate Judith Graham Pool, MD File photo courtesy of HANDI, NHF. Pool JG, Shannon AE. N Engl J Med. 1965: 273: 1443 -1447.
1960 s n n 1966: Hyland announces commercial availability of FVIII concentrates 1969: FIX concentrate licensed 1 1. Hoag MS, et al. N Eng J Med. 1969; 280(11): 581 -6.
1960 s n n n Continuous infusion Predictable and sustained level over time Cost savings due to decreased – Laboratory evaluations – Clearance over time Ideal replacement for surgical interventions and specific bleeding episodes in hemophilia Mc. Millan CW, et al. Br J Haematol. 1970; 18: 659 -667. Hathaway WE, et al. Am J Hematol. 1984; 17: 85 -88. Shulman NR, et al, Ann Intern Med. 1967; 67: 856.
1960 s 1969: First hemophilia camp established
1970 s n n n 1970 s: Home infusion therapy: a common treatment practice 1971: von Willebrand’s factor identified¹ 1974: First successful immune tolerance² 1975: Federally funded comprehensive hemophilia treatment centers initiated 1977: Mannucci discovers DDAVP increases FVIII and v. WF levels 3 1. 2. 3. Zimmerman, TS, et al. J Clin Invest. 1971; 50: 244 -254. Brackmann HH, Lancet 1977; 2 (8044): 933. Mannucci PM. Lancet. 1977; 1: 869 -872.
Regional Hemophilia Treatment Center Network
1970 s Revisiting use of Prophylaxis UK study – 9 boys with Hem A <1% - Randomized, blinded, crossover study - Had target joints present - Received 25% correction with FVIII or placebo once a week, followed by week of rest - Showed 15% reduction in bleeding episodes in 100 day period (from 13. 6 episodes to 11. 6) - 66% decrease in bleeds in first three days of the week. Aronstam. 1976. Brit J Haematol. 33: 81.
1970 s Transmission of blood-borne diseases: – Hepatitis B: HBV – Hepatitis non-A, non-B: Later called “C” – HIV Mannucci PM, et al. J Clin Pathol. 1975; 28(8): 620 -624. Schramm W, et al. Blut. 1989; 59(4): 390 -392.
Hepatitis B Virus: HBV n n n n Lipid-enveloped DNA virus Replicates within liver cells Transmitted by exchange of bodily fluids 90% recover with immunity; 10% develop chronic HBV of which 20 -30% progress to cirrhosis Virion sensitive to heat and solvent/detergent 1981: Hep B vaccine plasma derived 1987: Hep B vaccine recombinant licensed Electron micrograph of Hepatitis B Virions courtesy of the CDC
The Price of Independence Non-A, non-B (Hepatitis C) recognized as transmissible via plasma-derived products – 1986: Commercial screening for non-A, non-B hepatitis surrogate markers – 1989: Hepatitis C isolated – 1990: Hepatitis C Ab testing of donated blood begins
Hepatitis C Virus: HCV n Lipid-enveloped RNA virus n Replicates within infected liver cells n Transmitted by the exchange of bodily fluids n 85% or more with acute HCV infection progress to chronic hepatitis* n Virion sensitive to heat and solvent/detergent n No vaccine available * Seeff LB. Am J Med. Dec. 1999; 107[6 B]: 10 S-15 S.
1980 s MMWR July 16, 1982/31(27): 365 -367.
The Price of Independence n 1983: Suspicion that HIV threatened the worldwide blood supply April 1, 1983: Hemofil-T, first heat-treated FVIII concentrate in the U. S. 1984: Montagnier 1 and Gallo 2 discover HTLV-3 (HIV) 1984: Efficacy of heat treatment for viral inactivation demonstrated 1984: Recall of blood products initiated 1985: ELISA test used to detect HIV antibodies among blood donors n 1985: Safety net: n n n 1. 2. 3. Donor deferral Viral inactivation methods HIV antibody testing 1. Barre-Sinoussi F, et al. Science 1983; 220(4599): 868 -71. 2. Gallo RC, et al. Science 1984; 4; 224(4648): 500 -3.
The Heroes We Care For Photo courtesy of Mary Lou Cygan, R. N. , P. N. P.
1980 s
1990 s n 1991 – Karolinska University – Stockholm, Sweden Dr. Petrini and colleagues describe prevention of joint disease in boys with hemophilia under age two. Lusher, J. 2008. Hem. Aware. 13: 8.
1990 s HTC (%) Non-HTC (%) P Mild 21. 8 52. 8 <. 001 Moderate 24. 2 26. 7 Severe 54. 0 20. 5 6. 0 2. 3 <. 001 Characteristics Severity Inhibitors Liver disease 2. 3 0. 7 . 002 HIV infection 31. 1 17. 1 <. 001 AIDS 8. 2 5. 9 . 02 Soucie JM, et al. Blood. 2000; 96(2): 437 -442.
1990 s Mortality decreased 40% in patients using a comprehensive hemophilia treatment center (HTC) “The finding that HTCs have a significant effect on reducing mortality in patients with hemophilia supports the effectiveness of such centers in providing specialized preventative care. ” Soucie JM, et al. Blood. 2000; 96(2): 437 -442.
1990 s Prophylaxis recommendation n Primary – Early institution ~ 1 -2 years with the aim of maintaining trough > 1% – Longitudinal assessment of joint status essential n Secondary – Appropriate for intervention to prevent joint and other disease associated morbidities MASAC Recommendation #35. March 11, 1994.
2000 s Gene therapy trials begun n Joint Outcome Study published 1 n National Pain Survey n Willetabs and Wilbrintin advertised as treatment for v. WD n Manco-Johnson, et al. NEJM. 2007. Vol. 357: (6) 535 -544.
Hemophilia A: Evolution of Therapy Year Therapy 1950 Plasma (1 -FVIII U/ml) 1966 Cryoprecipitate (5 -FVIII U/ml) 1975 Lyophilized concentrates (30 -FVIII U/ml) 1983 Heat treatment of lyophilized concentrates for viral attenuation 1985 Introduction of a solvent detergent for viral inactivation 1988 Monoclonal antibody-purified FVIII concentrates with heat or solvent detergent treatment 1992 Recombinant DNA products: 1 st generation 2000 s 2 nd & 3 rd generation recombinant products
Hemophilia B: Evolution of Therapy Year Therapy 1950 Plasma (1 -FIX U/ml) 1975 Lyophilized prothrombin complex concentrates or PCC (30 -FIX U/ml) Heat treatment of lyophilized concentrates for viral attenuation Development of a solvent detergent for viral inactivation 1985 1992 Chromatographic/monoclonal antibody-purified FIX concentrate with ultrafiltration and thiocyanate treatment 1997 Recombinant DNA product
The Future n n n n Longer acting concentrates Recombinant therapy for v. WD Alternate route therapies Gene transplantation Elimination of transfusion-associated infections Understand overcome inhibitor development Quality of life issues – Elimination of joint morbidity – Optimizing the individual’s social and academic performance
The Future… …the promise of achieving your potential
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