Clinical trials evaluating pathogen-reduced platelet products: Methodologic issues and recommendations

Transfusion

Volumn 53, Issue 8, 2013, Pages 1843-1855

  Cook, Richard J ^a   Heddle, Nancy M ^a  

^a UNIVERSITY OF WATERLOO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AMOTOSALEN; RIBOFLAVIN;

CLINICAL TRIAL; HUMAN; PATHOGENESIS; REVIEW; RIGOR; SYNTHESIS; THROMBOCYTE TRANSFUSION; TREATMENT RESPONSE;

BLOOD SAFETY; HEMORRHAGE; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); PLATELET COUNT; PLATELET TRANSFUSION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN;

EID: 84882456507     PISSN: 00411132     EISSN: 15372995     Source Type: Journal    
DOI: 10.1111/j.1537-2995.2012.03951.x     Document Type: Review

References (35)

1. Guyatt G, Rennie D, Meade MO, Cook DJ,. Users′ guides to the medical literature: a manual for evidence-based clinical practice. 2nd ed. Chicago: McGraw-Hill Companies, Inc.; 2008.
2. Sackett DL, Haynes RB, Guyatt GH, Tugwell P,. Clinical epidemiology: a basic science for clinical medicine. 2nd ed. Boston (MA): Little, Brown and Company; 1991.
3. Meinert CL, Tonascia S,. Clinical trials: design, conduct and analysis. London: Oxford University Press; 1986.
4. van Rhenen D, Gulliksson H, Cazenave J-P, Pamphilon D, Ljungman P, Kluter H, Vermeij H, Kappers-Klunne M, de, Greef G, Laforet M, Lioure B, Davis K, Marblie S, Mayaudon V, Flament J, Conlan M, Lin L, Metzel P, Buchholz D, Corash L, euroSPRITE trial. Transfusion of pooled buffy coat platelet components prepared with photochemical pathogen inactivation treatment: the euroSPRITE trial. Blood 2003; 101: 2426-2433.
5. McCullough J, Vesole DH, Benjamin RJ, Slichter SJ, Pineda A, Snyder E, Stadtmauer EA, Lopez-Plaza I, Coutre S, Strauss RG, Goodnough LT, Fridey JL, Raife T, Cable R, Murphy S, Howard F 4th, Davis K, Lin JS, Metzel P, Corash L, Koutsoukos A, Lin L, Buchholz DH, Conlan MG,. Therapeutic efficacy and safety of platelets treated with a photochemical process for pathogen inactivation: the SPRINT Trial. Blood 2004; 104: 1534-1541.
6. Kerkhoffs JL, van Putten WL, Novotny VM, Te Boekhorst PA, Schipperus MR, Zwaginga JJ, van, Pampus LC, de, Greef GE, Luten M, Huijgens PC, Brand A, van, Rhenen DJ, Dutch-Belgian HOVON cooperative group. Clinical effectiveness of leucoreduced, pooled donor platelet concentrates, stored in plasma or additive solution with and without pathogen reduction. Br J Haematol 2010; 150: 209-217.
7. Cazenave J-P, Follea G, Bardiaux L, Boiron J-M, Lafeuillade B, Debost M, Lioure B, Harousseau J-L, Tabrizi R, Cahn J-Y, Michallet M, Ambruso D, Tissot JD, Sensebé L, Kondo T, McCullough J, Rebulla P, Escolar G, Mintz P, Heddle NM, Goodrich RP, Bruhwyler J, Le C, Cook RJ, Deberdt L, et al. A randomized controlled clinical trial evaluating the performance and safety of platelets treated with MIRASOL pathogen reduction technology. Transfusion 2010; 50: 2362-2375.
8. Janetzko K, Cazenave JP, Kluter H, Kientz D, Michel M, Beris P, Lioure B, Hastka J, Marblie S, Mayaudon V, Lin L, Lin JS, Conlan MG, Flament J,. Therapeutic efficacy and safety of photochemically treated apheresis platelets processed with an optimized integrated set. Transfusion 2005; 45: 1443-1452.
9. Lozano M, Knutson F, Tardivel R, Cid J, Maymo RM, Lof H, Roddie H, Pelly J, Docherty A, Sherman C, Lin L, Propst M, Corash L, Prowse C,. A multi-centre study of therapeutic efficacy and safety of platelet components treated with amotosalen and ultraviolet A pathogen inactivation stored for 6 or 7 d prior to transfusion. Br J Haematol 2011; 153: 393-401.
10. European Directorate for the Quality of Medicines & HealthCare, Council of Europe. Executive Summary: Symposium on Implementation of Pathogen Reduction Technologies for Blood Components: European Comittee (Partial Agreement) on Blood Transfusion (CD-P-TS). 2011. [cited 2012 Nov 2]. Available from: URL: http://www.edqm.eu/en/healthcare-news-44.html
11. Heddle NM,. The research question. Transfusion 2007; 47: 15-17.
12. Sackett DL, Gent M,. Controversy in counting and attributing events in clinical trials. N Engl J Med 1979; 301: 1410-1412.
13. Rebulla P, Finazzi G, Marangoni F, Avvisati G, Gugliotta L, Tognoni G, Barbui T, Mandelli F, Sirchia G,; Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto. The threshold for prophylactic platelet transfusions in adults with acute myeloid leukemia. N Engl J Med 1997; 337: 1870-1875.
14. Heddle NM, Cook RJ, Tinmouth A, Kouroukis CT, Hervig T, Klapper E, Brandwein JM, Szczepiorkowski ZM, AuBuchon JP, Barty RL, Lee KA,; SToP Study Investigators of the BEST Collaborative. A randomized controlled trial comparing standard- and low-dose strategies for transfusion of platelets (SToP) to patients with thrombocytopenia. Blood 2009; 113: 1564-1573.
15. Slichter SJ, Kaufman RM, Assmann SF, McCullough J, Triulzi DJ, Strauss RG, Gernsheimer TB, Ness PM, Brecher ME, Josephson CD, Konkle BA, Woodson RD, Ortel TL, Hillyer CD, Skerrett DL, McCrae KR, Sloan SR, Uhl L, George JN, Aquino VM, Manno CS, McFarland JG, Hess JR, Leissinger C, Granger S,. Dose of prophylactic platelet transfusions and prevention of hemorrhage. N Engl J Med 2010; 362: 600-613.
16. Cook RJ, Heddle NM, Rebulla P, Sigouin CS, Webert KE,. Methods for the analysis of bleeding outcomes in randomized trials of PLT transfusion triggers. Transfusion 2004; 44: 1135-1142.
17. Mullahy J, Manning W,. Statistical issues of cost-effectiveness analysis. Cambridge: Cambridge University Press; 1994.
18. Bishop JF, Matthews JP, Yuen K, McGrath K, Wolf MM, Szer J,. The definition of refractoriness to platelet transfusions. Transfus Med 1992; 2: 35-41.
19. Heddle NM, Cook RJ, Blajchman MA, Barty RL, Sigouin CS, Boye DM, Nelson EJ, Kelton JG,. Assessing the effectiveness of whole blood-derived platelets stored as a pool: a randomized block noninferiority trial. Transfusion 2005; 45: 896-903.
20. Heddle NM, Arnold DM, Webert KE,. Time to rethink clinically important outcomes in platelet transfusion trials. Transfusion 2011; 51: 430-434.
21. Heddle NM, Cook RJ,. Composite outcomes in clinical trials: what are they and when should they be used? Transfusion 2011; 51: 11-13.
22. Prentice RL,. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med 1989; 8: 431-440.
23. Webert KE, Arnold DM, Lui Y, Carruthers J, Arnold E, Heddle NM,. A new tool to assess bleeding severity in patients with chemotherapy-induced thrombocytopenia. Transfusion 2012; 52: 2466-2474.
24. Cazenave JP, Davis K, Corash L,. Design of clinical trials to evaluate the efficacy of platelet transfusion: the euroSPRITE trial for components treated with Helinx technology. Semin Hematol 2001; 38 (Suppl 11): 46-54.
25. Slichter SJ, Davis K, Enright H, Braine H, Gernsheimer T, Kao KJ, Kickler T, Lee E, McFarland J, McCullough J, Rodey G, Schiffer CA, Woodson R., Factors affecting posttransfusion platelet increments, platelet refractoriness, and platelet transfusion intervals in thrombocytopenic patients. Blood 2005; 105: 4106-4114.
26. Yusuf S, Wittes J, Probstfield J, Tyroler HA,. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. JAMA 1991; 266: 93-98.
27. Pullenayegum EM, Cook RJ,. The analysis of treatment effects for recurring episodic conditions. Stat Med 2010; 29: 1539-1558.
28. Cook R, Lawless J,. The statistical analysis of recurrent events. New York: Springer; 2007.
29. Andersen PK, Gill RD,. Cox's regression model for counting processes: A large sample study. Ann Stat 1982; 10: 1100-1120.
30. Lawless JF, Nadeau JC,. Nonparametric estimation of cumulative mean functions for recurrent events. Technometrics 1995; 37: 158-168.
31. Cook RJ, Lawless JF, Nadeau C,. Robust tests for treatment comparisons based on recurrent event responses. Biometrics 1996; 52: 557-571.
32. Therneau TM, Grambsch PM,. Modeling survival data: extending the Cox model. New York: Springer; 2004.
33. McCulloch CE, Searle SR,. Generalized, linear, and mixed models. New York: John Wiley & Sons; 2005.
34. Liang K-Y, Zeger SL,. Longitudinal data analysis using generalized linear models. Biometrika 1986; 73: 13-22.
35. Vamvakas EC,. Rationale for randomized controlled trials and for intention-to-treat analysis in transfusion medicine: are they one and the same? Vox Sang 2008; 95: 165-173.