National needs assessment of patients treated at the United States Federally-Funded Hemophilia Treatment Centers.

AIM: The National Hemophilia Program Coordinating Center, with the U.S. Regional Hemophilia Network conducted a national needs assessment of U.S. Hemophilia Treatment Center (HTC) patients. The objectives were to determine: (i) To what extent do patients report that they receive needed services and education; (ii) How well do the services provided meet their needs; and (iii) What are the patients' perspectives about their care. METHODS: A survey was mailed to active patients of 129 HTCs. Respondents completed the anonymous surveys on line or returned them by mail. Questions focused on management and information, access and barriers to care, coping, resources, and transition. RESULTS: Of 24 308 questionnaires mailed, 4004 (16.5%) were returned. Most respondents reported very few gaps in needed services or information and reported that services and information met their needs. Over 90% agreed or strongly agreed that care was patient-centred and rated HTC care as important or very important. Identified gaps included dietary advice, genetic testing, information on ageing, sexual health and basic needs resources. Minority respondents reported more barriers. CONCLUSION: This survey is the largest assessment of the HTC population. Respondents reported that the services and information provided by the HTCs met their needs. Quality improvement opportunities include transition and services related to ageing and sexual health. Further investigation of barriers to care for minorities is underway. Results will help develop national priorities to better serve all patients in the US. HTCs.

Cerebral hemisphere asymmetry in CT and functional recovery from hemiplegia.

Cerebral hemispheric asymmetries were assessed in relation to motor and functional recovery in nine patients with stroke. All were globally aphasic, assuring similar location and extent of lesion. Initial motor and functional ability were appraised from medical records and compared with neurologic and functional outcome. Frontal and occipital hemispheric widths and lengths were determined from CT. Patients were classified into three groups on the basis of mean combined asymmetry for width (typical asymmetry, atypical asymmetry, and equal symmetry). Patients with the most atypical cerebral asymmetries showed greater recovery than patients in other groups. Two patients with atypical mean occipital asymmetry (width) fared best functionally, whereas the patient who showed the greatest motor recovery had the most atypical mean occipital asymmetry (width). Cerebral asymmetries may contribute to recovery after stroke.

A model for multisystem evaluation treatment of individuals with Parkinson's disease.

In this article, we present a model of Parkinson's disease based on pathophysiology, impairments, and disabilities. The model provides a format for deciding what to assess, for interpreting assessment findings, and for deciding which impairments can be corrected by physical therapy intervention and which cannot. From the model, we can predict effective treatment approaches. We can also use the model to identify specific research questions related to the efficacy of physical therapy intervention.

A model for multisystem evaluation, interpretation, and treatment of individuals with neurologic dysfunction.

We have presented a model that can be used to organize current concepts of neuroanatomy, neurophysiology, and motor control theory into a clinically relevant context. This model can be used to postulate the causal relationship among pathology, impairments, and disabilities. Knowledge of the insult often provides predictive information regarding stability of the disability, progression of the disease, and possibly recovery. Knowledge of neuropathology should guide the clinician regarding the types of impairments that are expected. Thus, by knowing the type and location of the lesion, the clinician already has a perspective regarding which evaluative tests should be emphasized and what treatment strategies will be most effective. In the absence of known pathology, knowledge of motor control theory, neuroanatomy, and neuropathology can still help the clinician make educated judgments regarding the interrelationship of the patient's impairments. In this model, we separate impairments that result directly from neuropathology and those that arise indirectly to help the clinician keep the total patient in perspective and to focus intervention. This separation emphasizes the applicability and limitations of motor control theories in the treatment of neurologically impaired individuals. Motor control theory helps to identify the relationship between direct impairments and their likely consequences; motor control theory does not address the very real issue of impairments that arise indirectly in systems such as the musculoskeletal and cardiopulmonary systems. Impairments that occur as a composite effect of multiple causes are identified as a separate category. Some of the causes of these impairments can often be addressed by physical therapy intervention, and some cannot.(ABSTRACT TRUNCATED AT 250 WORDS)

Management of individuals with Parkinson's disease: rationale and case studies.

In this article, we present a systematic approach to physical therapy management of individuals with Parkinson's disease. This approach is based on a model that relates knowledge of the underlying pathology of the disease to impairments and disability. We discuss use of the model to evaluate, interpret, and treat impairments and disabilities of the patient with Parkinson's disease. We emphasize the relative influence of impairments that arise directly from the neuroanatomical pathology and those that arise indirectly through subsequent musculoskeletal alterations. We illustrate the use of the model in setting goals and in developing a treatment program. Two case studies are presented to illustrate the application of this approach to specific patient care. These case studies suggest the efficacy of physical therapy initiated early in the disease process. The first case study illustrates improvements of balance, gait, and functional movement made by an individual who was not yet receiving medication for Parkinson's disease. The second case study illustrates improvements of balance, gait, and functional movement made by an individual who was already receiving medication for the disease. These case studies illustrate the dramatic improvements that can be achieved in the patient with Parkinson's disease.

Understanding the basics of beta thalassemia major.

beta thalassemia major, a severe genetic disorder of the red blood cell, affects about 1,400 people in the United States, including children. Clinical management of this disorder has improved significantly in recent years, and ongoing research will lead to an even brighter outlook in the future.

Low recovery in vivo of highly purified factor VIII in patients with hemophilia.

To assess whether the response and recovery of highly purified factor VIII products approach the levels predicted by use of manufacturers' suggestions, we studied response in vivo and percentage of recovery in boys and men with hemophilia after infusion of 50 IU of an immunoaffinity-purified factor VIII preparation (Hemofil M) per kilogram of body weight. We also studied dose response and percentage of recovery in the same boys after infusion of a factor VII concentrate prepared by solvent detergent treatment and gel filtration (Koate-HP). For boys the difference between mean peak factor VIII response with Hemofil M (1.02% +/- 0.07%) and the response with Koate-HP (1.21% +/- 0.10%) was not significant (p = 0.10), but the response of both products was considerably less than the predicted response of 2.0%. The response to Hemofil M in men (1.38% +/- 0.12%) was significantly better than the response in boys (p = 0.004) but, again, fell short of the anticipated response of 2.0%. In boys the percentage of recovery of Hemofil M was 38.4% +/- 2.1%, compared with Koate-HP recovery, which was 47.0% +/- 3.6% (p = 0.034). The percentage of recovery in men was 47.5% +/- 2.8%. The better response observed in men appears to be, in part, a function of larger body size. We conclude that peak factor VIII response and recovery in boys and men who receive highly purified factor VIII concentrates are lower than those predicted by use of current prescribing recommendations.

Loss of detectable antibody to hepatitis B surface antigen in immunized patients with hemophilia but without human immunodeficiency virus infection.

We investigated the loss of detectable antibody directed against the hepatitis B surface antigen after immunization with hepatitis B vaccine in 55 patients who were seronegative for the human immunodeficiency virus and had hemophilia. Twenty percent of patients lost detectable antibody at a mean of 4.6 years after completion of immunization. Monitoring of hepatitis B serostatus or routine revaccination may be necessary.

Headache in cerebral embolic disease.

A patient is presented who had severe headache and then developed a stuttering course of clinical neurological deficits. Arteriography demonstrated an ipsilateral proximal middle cerebral embolus. With recurrence of symptoms, repeat arteriography showed another, more proximal embolus with areas of distal occlusion. The headache probably occurred when the embolus became lodged in a pain sensitive cerebral vessel at the base of the brain. The subsequent stuttering neurological deficits were due to fragmentation of the embolus as well as recurrence.

Complex regional pain syndrome in pediatric patients with severe factor VIII deficiency.

Two boys with severe factor VIII deficiency that initially presented with acute onset of joint pain and swelling consistent with an uncomplicated hemarthrosis are reported. When appropriate management failed to provide resolution of symptoms, alternate diagnoses were considered. Both boys ultimately had complex regional pain syndrome (CRPS) diagnosed. The delay in diagnosis contributed to prolonged patient discomfort and lack of appropriate therapy. Complex regional pain syndrome encompasses a group of disorders that are characterized by pain severity or duration disproportionate to that expected. It is uncommon in the pediatric population. Because early diagnosis and appropriate treatment may improve outcome, it is important for practitioners to consider CRPS in the differential diagnosis of persistent pain in children with hemophilia.

Beta-thalassemia major and sickle cell disease.

Beta-thalassemia major and sickle cell disease are genetic disorders of red blood cells, caused by abnormal hemoglobin. These hemoglobinopathies affect males and females equally. Both are chronic disorders requiring lifelong treatment for affected individuals and education and support for them and their families. Special nursing considerations for the care of women facing the unique challenges of these disorders will be discussed.

Superior in vivo response of recombinant factor VIII concentrate in children with hemophilia A.

OBJECTIVE: Our previous experience with highly purified plasma-derived factor VIII (pdFVIII) concentrates showed that adult dosage recommendations were not applicable to children. In this study, we compared the in vivo response and recovery of recombinant factor VIII (rFVIII) with those of highly purified pdFVIII concentrate in children with hemophilia A. STUDY DESIGN: Ten boys with severe factor VIII deficiency and no concurrent bleeding episodes participated in a masked, prospective, crossover study comparing factor VIII coagulant activity after infusion of 50 units of pdFVIII and rFVIII products per kilogram of body weight. RESULTS: Mean peak factor VIII response with rFVIII was 1.91% +/- 0.14%, significantly better than the response observed with highly purified pdFVIII of 1.5% +/- 0.15% (p = 0.007). Mean peak factor VIII recovery was 100.5% with rFVIII versus 78.7% with pdFVIII (p = 0.007). Positive correlations between response to rFVIII and body surface area (r = 0.734, p = 0.015), body weight (r = 0.762, p = 0.01), and plasma volume (r = 0.659, p = 0.03) were observed. CONCLUSIONS: Infusion of rFVIII produced a significantly better response and recovery in vivo than infusion of highly purified pdFVIII in children. The response in children after infusion of rFVIII was similar to the response previously observed in adults.

Developing theory-based risk-reduction interventions for HIV-positive young people with haemophilia.

Eleven haemophilia treatment centres in the United States collaborated in the Hemophilia Behavioural Intervention Evaluation Projects (HBIEP) to develop theory-based interventions to reduce the risk of HIV transmission from seropositive adolescents and young adults with haemophilia. While the Transtheoretical Model of Behaviour Change and the Theory of Reasoned Action provided the theoretical underpinnings, the exact form in which these theories would be applied depended on developmental research. This paper presents the various phases of the process to develop the theory based interventions: literature review, qualitative interviews, quantitative surveys, a provider survey, a materials review, and the actual planning. All or portions of this process could be applied to the development of interventions for many behaviour-change projects. A description of the HBIEP interventions is also provided.