Abstract
We evaluated the number and characteristics of randomized controlled trials (RCTs) addressing hematopoietic stem cell transplantation (HSCT) for patients with hematological malignancies, comparing the productivity of US and Europe. A MEDLINE search was conducted to identify all published RCTs for the management of adult patients with hematological malignancies from January 1992 to December 2003. Eighty-three of the 306 trials identified included HSCT as one of the treatment arms. The US produced 25, Europe 54, and all other countries four. Four European countries, France, Italy, Germany, and UK (FIGU), produced 32 out of the 54 European studies. Significant differences emerged when focus of the study and accrual numbers were analyzed. Trials comparing HSCT to standard dose therapy represented 34.9% of the 83 trials and 59.4% of FIGU trials, but only 4% of US studies (P=0.001). US trials accrued a mean of 110.2 patients per study, as compared to 222.6 in FIGU studies (P=0.006) and 205.3 when all non-US countries are considered (P=0.01). Our conclusions are that US transplant RCT have focused on issues other than the comparison of HSCT to standard therapies. There is serious paucity of US trials defining the role of HSCT in the management of hematological malignancies.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Simon R . Design and Analysis of Clinical Trials. In: De Vita V, Hellman S, Rosemberg SA (eds). Cancer: Principles & Practice, 6th edn. Lippincott Williams & Wilkins: Philadelphia, PA, 2001, pp 521–538.
Preventive Services Task Force. Guide to Clinical Preventive Services: Report of the U.S. Preventive Task Force, 2nd edn. Williams & Wilkins: Baltimore, MD, 1996.
Stossel TP, Stossel SG . Declining American representation in leading Clinical-Research Journals. N Engl J Med 1990; 322: 739–742.
Friedman MA, Cain DF, Bronzert D, Wu RS . Poor funding rates of cancer clinical research: intractable problem or solvable challenge? J Nat Cancer Inst 1991; 83: 838–841.
Freireich EJ . A study of the status of clinical cancer research in the United States. J Natl Cancer Inst 1991; 83: 829–837.
Cassileth BR . Clinical trials: time for action. J Clin Oncol 2003; 21: 765–766.
O'Reilly RJ . Clinical trials of hematopoietic cell transplantation: current needs and future strategies. Biol Blood Marrow Transplant 2000; 6: 79–89.
Hillner BE . Trends in clinical trials reports in common cancers between 1989 and 2000. J Clin Oncol 2003; 21: 1850–1858.
Dickersin K, Scherer R, Lefebvre C . Systematic reviews: identifying relevant studies for systematic reviews. BMJ 1994; 309: 1286–1291.
International Bone Marrow Transplant Registry/American Bone Marrow Transplant Registry, Annual Publication, 2003.
Gratwohl A, Schmid O, Baldomero H, Horisberger B, Urbano-Ispizua A . Haematopoietic Stem Cell Transplantation (HSCT) in Europe 2002. Changes in indications and impact of team density. A report of the EBMT activity survey. Bone Marrow Transplant 2004; 34: 855–875.
Wright JR, Whelan TJ, Schiff S, Dubois S, Crooks D, Haines PT et al. Why cancer patients enter randomized clinical trials: exploring the factors that influence their decision. J Clin Oncol 2004; 22: 4312–4318.
Comis RL, Miller JD, Aldige CR, Krebs L, Stoval E . Public attitudes toward participation in cancer clinical trials. J Clin Oncol 2003; 21: 830–835.
Scherer FM . The pharmaceutical industry-prices and progress. N Engl J Med 2004; 351: 927–932.
Bodenheimer T . Uneasy alliance. Clinical investigators and the pharmaceutical industry. N Engl J Med 2000; 342: 1539–1544.
Gross CP, Murthy V, Li Y, Kaluzny AD, Krumholz HM . Cancer trial enrollment after state-mandated reimbursement. J Natl Cancer Inst 2004; 96: 1063–1069.
Corrie P, Shaw J, Harris R . Rate limiting factors in recruitment of patients to clinical trials in cancer research: descriptive study. BMJ 2003; 327: 320–321.
Natham D, Varmus HE . The national institutes of health and clinical research: a progress report. Nat Med 2000; 6: 1202–1204.
Natham DG, Wilson JD . Clinical research and the NIH-A report card. N Engl J Med 2003; 349: 1860–1865.
Crowley WF, Sherwood L, Salber P, Scheinberg D, Slavkin H, Tilson H et al. Clinical research in the United States at a crossroads: proposal for a novel public–private partnership to establish a National Clinical Research Enterprise. JAMA 2004; 291: 1120–1126.
Author information
Authors and Affiliations
Corresponding author
Rights and permissions
About this article
Cite this article
Kuthiala, S., Lyman, G. & Ballester, O. Randomized clinical trials for hematopoietic stem cell transplantation: lessons to be learned from the European experience. Bone Marrow Transplant 37, 219–221 (2006). https://doi.org/10.1038/sj.bmt.1705230
Received:
Revised:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1705230