Decreased morbidity and mortality of autologous hematopoietic transplants for children with malignant central nervous system tumors: the 'Head Start' trials, 1991-2009.

Since 1991, three sequential prospective clinical trials have been conducted by the 'Head Start' (HS) Consortium in which young children with newly-diagnosed malignant central nervous system (CNS) tumors were treated with induction chemotherapy followed by single-cycle marrow-ablative chemotherapy and autologous hematopoietic rescue as a means of improving disease cure rate and quality of survival through avoidance (<6 years old at diagnosis) or reduction (6-10 years old) of brain irradiation. Bone Marrow (HS I) or filgrastim-mobilized peripheral hematopoietic cells (HS II and III) were obtained following recovery from the first and/or second induction cycles. Radiotherapy was administered following all chemotherapy only for patients with residual tumor following completion of induction or with age greater than 6 years at diagnosis. Two hundred and twenty-six children were enrolled on three consecutive HS trials with primary malignant CNS tumors and underwent marrow-ablative chemotherapy. The 100-day treatment-related mortality (TRM) steadily declined as did grade IV transplant-related oropharyngeal mucositis. Factors most likely associated with the decrease in TRM and morbidity are increasing experience with the marrow-ablative chemotherapy regimen combined with improved leukapheresis and post-reinfusion supportive care techniques, contributing toward improved overall survival.

Data utilization, not data acquisition, is the main problem.

Previous studies focusing on the value of laboratory testing have sometimes been flawed because they had not always determined whether the data generated had been appropriately used by clinicians. It has been well documented that neither clinicians nor administrators always use objective data effectively. When data are not used appropriately by clinicians, not only are morbidity and mortality increased, but also care costs are higher. Failures by administrators result in lost opportunities to identify and correct system deficiencies. To provide information support to assure good data utilization, a prototype desktop workstation has been developed and partially implemented. Experience with such a system suggests that not only may it help provide better care more economically, but it can also aid in implementation of Practice Guidelines (Agency for Health Care Policy and Research) and Clinical Indicator (Joint Commission on Accreditation of Healthcare Organizations) programs. Laboratorians can be influential in this effort and promote the use of new knowledge in patient care in a timely and responsible manner.

Use of comprehensive laboratory data as a management tool.

Studies of the use of laboratory data in cost-control management yield the following conclusions: (1) Comprehensive medical information is a valuable medical resource; (2) problems in practice frequently involve poor data utilization, and difficulties with data acquisition are frequently secondary phenomena; (3) current regulations limiting the acquisition of data may be counterproductive in worsening care while raising costs. If continued, the long-term effects on knowledge generation can be expected to be devastating; (4) good medicine is cost effective; (5) poor medicine is expensive, both in human and economic terms; and (6) good medical care requires a patient-oriented data base, a clinical decision-support system, a feed-back mechanism so that effectiveness of the system may be evaluated on an ongoing basis, and adequately trained medical technology personnel.

Building a data base for monitoring and facilitating health care.

Our experience suggests that (1) comprehensive objective information is a valuable medical resource; (2) the systematic acquisition of objective information in a form suitable for computers is a necessary first step to building a useful data base; (3) when appropriately utilized, objective medical information may permit a study of medical practice, cost of care, and operational efficiency; (4) analysis of costs divorced from outcome and rehospitalization experiences may be misleading; (5) for some desired studies, the information required may be obtainable within a single institution. For others, however, community-wide studies may be the only possible way to obtain the information needed; (6) useful computer systems will be developed only if the health professionals become more knowledgeable (and demanding) and administrators are persuaded to provide effective support; and (7) effective hospital support for adequate medical information systems will probably come when criteria evaluating hospital performance are altered to reflect society's needs. Our experience with computer evaluation of care has been a sobering one. Still, it is obvious that the technology offers considerable hope that many inefficiencies in practice can be corrected. Considering all the current jeremiads about what we can afford in health care, it is reassuring to learn that good medicine is cost effective.

Cost effectiveness of multiphasic screening: old controversies and a new rationale.

The cost effectiveness of multiphasic screening is evaluated from a conceptual as well as from a practical viewpoint. Conceptual analysis includes a consideration of the technical sensitivity and specificity of the tests used, of the prevalences of the screened diseases, and of the costs and values associated with different outcomes of screening. Practical considerations include the potential of multiphasic screening for increasing productivity, for reassuring patients, and for reducing morbidity and mortality. Although all these issues can be cogently formulated, at present none can be documented by a comprehensive set of data leading to irrefutable conclusions. Therefore, the issue of who should be screened continues to be obfuscated by controversy and prejudice. To resolve this dilemma a new rationale for the use of multiphasic screening is developed. This is based on a small number of uncontroversial facts and leads to practical proposals relating to how standards for useful test batteries can be constructed.

Automated donor-recipient identification systems as a means of reducing human error in blood transfusion.

Clerical and administrative errors are the most common mistakes in blood banking, with errors of sample or patient identification accounting for the majority of mistakes. In an effort to reduce the incidence of human errors, three automated donor-recipient identification systems were developed and were evaluated under clinical conditions. After two years of development and bench testing, each set of equipment was tested, along with established procedures, at a large clinical center, a medium-sized community hospital and a regional donor center. Although none of the systems, as tested, proved acceptable under clinical conditions, a great deal was learned about prototype equipment testing in the clinical setting. Future development of identification systems for clinical use should respond to carefully defined problems, apply in all areas of the hospital or donor center, and be convenient and reliable. The development of the equipment should involve established manufacturers and be subject to exacting preclinical testing.