Objective:
To assess treatment of young adult (YA) patients with hemophilia (PWH) and issues around access to and use of factor and comprehensive care.
Methods:
Analysis of US respondents aged 18-30 years in the international HERO study conducted in 2010-2011.
Summary:
Of 189 adult PWH HERO respondents in the United States, 66 were aged 18-30 years. More YA-PWH were on prophylaxis (50%) than on-demand alone (24%) or with occasional short-term prophylaxis (24%). Only 27% used treatment medication exactly as prescribed, with 27% a little less, and 21% varying (sometimes more/less). Twenty-six percent reported issues with access to factor in the prior 5 years due to availability or affordability, with 82% citing financial issues. YA-PWH reported a median of 2 bleeds in the prior month and median (IQR) annual bleed rate of 9.5 (12-22) bleeds. Similar to older adults, 80% of YA-PWH reported that a single joint suffers more bleeds than others; the most common joints reported were the ankle (77%), elbow (26%), and knee (21%). YA-PWH rated perceived disease control (1-10 scale, 10=most control) as median (IQR) 8 (7-9). HTC visit frequency was median (mean) 1 (1.66) per year. Similar to older adults (aged >40 years), 21% of YA-PWH reported difficulty in visiting the HTC. Accessibility was the most common reason (79%)—distance to travel (57%) or time to travel (29%); time constraints were more commonly reported by YA-PWH (57%), including being unable to get time off work (50%). When identifying health care professionals (HCPs) involved in management of their hemophilia, YA-PWH named hematologists (83%), nurses (67%), social workers (48%), counselors/psychologists (30%), and physical therapists (26%). The majority were satisfied with care provided by HCPs. YA-PWH reported being very/somewhat knowledgeable about hemophilia (91%). When looking to the future (pessimistic=1 to optimistic=7), YA-PWH were generally optimistic, with median (IQR) 5 (5-7).
Conclusions:
YA-PWH in the United States are more likely to be on prophylaxis than older adults, but less likely to use medication exactly as prescribed. Additional research is warranted to better understand why prescribed regimens are not followed. Despite 50% prophylaxis use, bleeding occurred ~1-2 times/month and YA-PWH reported high perceived disease control. During this period of transition to independence, it is important to note one quarter reported issues around access to treatment and one fifth reported difficulty in visiting the HTC. YA-PWH were satisfied with care, but infrequently reported social workers and physical therapists as part of management.
Objective:
Patient health outcomes are strongly correlated with management of their health condition. In chronic disease management, example hemophilia, a clinician often encounters situations where despite what the patient is instructed to do, there is resistance to follow directions, thus compromising the potential benefits of their treatment regimen.
The objective of these interventions was to test out motivational interviewing (MI) as an alternative communication approach to traditional advice -giving in especially difficult, yet common, hemophilia patient situations.
Methods:
Four case studies are reported, each with a different nurse, patient, and desired behavioral change. In each case study, the clinician had received education on the use of MI in health care settings. Multiple Tools and Paradigms were used with patients at different life stages, and in need of making changes in their self-care and disease management. The clinicians avoided traditional directive approaches and adopted collaborative methods that guided the patient to take responsibility for achieving their own health goals. Patients were followed post intervention to determine longevity of the success achieved through this intervention.
Results Summary:
In each case, the use of MI enabled the clinician to work collaboratively with the patient or caregiver, to evoke their own reasons for change, to elicit change planning, and to mobilize them towards healthier choices in managing their condition.
Case study 1: pediatric setting - Use of Engaging MicroskillsMicro skills (Open Questions, Affirmations, Reflections, Summaries) with a parent to empower their child to start self infusion.
Case study 2: pediatric setting - Use of MI Rulers to motivate an adolescent to choose a more appropriate sport activity.
Case study 3: Transition setting - Use of MI Spirit, especially partnership and honoring autonomy, with an adolescent.
Case study 4: Adult setting - Use of EPE (Elicit Provide Elicit Approach) with an adult who was not adherent to his prophylaxis regimen.
Conclusions:
MI was confirmed as a successful therapeutic approach with a variety of resilient clinical cases where traditional directive approaches had been unsuccessful. The use of MI created an open and trusted communication channel between the clinician and the patient or caregiver, concluding with a change plan agreement. Clinicians felt more fulfilled with their jobs and more satisfied with the result of their intervention. With appropriate education about this methodology, clinicians can use it with their patients where a behavioral change is required to achieve better therapeutic outcomes.
Objective:
Joint status was assessed in the 3-year SPINART study, which compared routine prophylaxis versus on-demand treatment in adults with severe hemophilia A. We report joint outcomes at year 3 obtained using magnetic resonance imaging (MRI).
Methods:
The open-label, randomized, controlled, parallel-group, multinational SPINART study enrolled males aged 12–50 years with severe hemophilia A who had ≥150 exposure days with any factor VIII (FVIII) product, no inhibitors, no prophylaxis for >12 consecutive months in the past 5 years, and 6–24 documented bleeding events or treatments in the previous 6 months. Patients were treated with Bayer’s sucrose-formulated recombinant FVIII (rFVIII-FS), either on demand or as prophylaxis (25 IU/kg 3 times weekly, with dose escalation by 5 IU/kg permitted once per year). MRI was performed at baseline and year 3 to evaluate the structure of 6 index joints. Each MRI was read by 3 radiologists blinded to treatment assignment who independently completed the extended MRI (eMRI) scale; higher eMRI scores indicate greater joint structure damage. The score for each joint was based on the readers’ median change score for each of the 45 eMRI scale items when comparing MRIs from different timepoints. Total patient score was derived; change from baseline in total patient score was prespecified as the first in a hierarchy of 2 secondary endpoints. Between- group comparison was made using constrained longitudinal data analysis. Data are presented for the intent-to-treat population.
Summary:
Of 84 patients enrolled (42 per treatment group), MRI data were available for 38 on-demand and 41 prophylaxis patients. Least squares (LS) mean change from baseline to year 3 on the eMRI scale total score was 0.96 for on demand and 0.79 for prophylaxis (LS mean difference, –0.17; 95% CI, –0.92 to 0.59; P=0.66). LS mean change from baseline to year 3 for on demand and prophylaxis was 0.06 and 0.01 for the eMRI soft-tissue domain (LS mean difference, –0.04; 95% CI, –0.18 to 0.10; P=0.53) and 0.90 and 0.78 for the eMRI osteochondral domain (LS mean difference, –0.12; 95% CI, –0.82 to 0.58; P=0.74).
Conclusions:
In adults with severe hemophilia A, progression of structural joint damage was not significantly different between patients using rFVIII prophylactically or on demand over a 3-year follow-up period, although less progression was seen with prophylaxis.
The purpose of this project is to determine if children with hemophilia have gross motor delays. Gross motor skills include, but are not limited to walking, running, jumping, climbing, crawling, balancing, kicking, catching and throwing activities. The large muscles of the body are responsible for performing these types of activities. Strength, balance and coordination are needed to demonstrate and improve these skills. Children with hemophilia may experience internal bleeding in their joints or muscles, which limits their activity. Some children with hemophilia may be restricted from active play or sports for fear of getting an injury that could cause internal bleeding. When activity is restricted, there can be decrease in strength, balance and endurance. If a child has gross motor delays, it puts him at risk for injury when playing with his peers. Physical Therapy evaluations in the Hemophilia Treatment Center help determine changes caused by bleeding episodes. There are specific motor skills children master as they grow, which represent strength, balance and coordination. The comprehensive clinic visits do not allow time to complete an intensive gross motor assessment. Physical Therapists use Manual Muscle Testing, MMT, to grade the strength of each muscle group. MMT strength testing is not appropriate for young children and does not represent strength during functional activities. To accurately determine children's muscle strengths, a standardized gross motor test should be used. The PT at the Comprehensive Care Center for Inherited Blood Disorders will conduct gross motor evaluations in conjunction with the annual visit. The scores will be evaluated to determine which patients have gross motor delays, so they can be referred for therapy services. The BOT 2 is the standardized gross motor test that will be used to determine gross motor levels. The gross motor skills that will be evaluated include bilateral coordination, balance, running speed and agility, upper limb coordination and strength. All patients with hemophilia between the ages of 4 and 12 years will be eligible to be evaluated in the upcoming year with additional PT testing with their annual visit. Patients may be referred to therapy or given a home exercise program depending on the deficits noted during the assessments. The goal is to improve our standard of care at the HTC, by adding gross motor screening for our patients to ensure appropriate referrals are made for therapy services.
