Objective:
When a surgical procedure is required in a patient with hemophilia, continuous infusion of factor (CIF) is a safe and effective alternative to bolus dosing.1-5 However, when cultural values collide with best practices, a patient-centered collaborative care plan is necessary to help ensure a positive outcome while respecting the core values of the patient.
Method:
Our team collaborated with our local Hemophilia Treatment Center (HTC) physicians and nurses to plan CIF for an Amish patient who required a total knee replacement. After interviewing the patient, the care team recognized when the patient transitioned on CIF to the home, we would need to respect the cultural beliefs of the patient without compromising the care.
The HTC physician and nurse ordered continuous infusion of factor for the patient with goal factor levels to remain between 70-100% on post-op days 1-7 and between 50-70% on post- op days 8-14. The home infusion team collaborated with the family and HTC team to finalize the care plan. The patient-centered decision prompted the use of a battery powered ambulatory infusion pump and the use of pre-approved sliding scale factor orders (ACAT protocol) with daily factor levels. Our biggest barrier was in respect to communication with the patient. In our local Amish community, telephones are not accessible. Our brainstorming lead to our COO suggesting we include a battery operated phone that would be attached to the pump and only be used for pump emergencies.
Summary:
The patient was discharged to home post-op day three with recombinant factor VIII running at 2.5 units/kg/hour via a battery operated ambulatory pump. Levels on post-op day four were below 70, prompting the nurse to call on the “pump phone” and return for a visit that night to increase the rate to 2.7 units/kg/hour. The rate remained the same throughout the remainder of the therapy and the levels stayed within the range designated by the physician.
Conclusion:
The patient-centered multidisciplinary care plan allowed for a positive outcome while respecting the patient’s culture.
Objective:
This prospective clinical trial was conducted to assess the safety, efficacy and PK of BAX326 (a novel recombinant FIX [rFIX] manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps [solvent/detergent treatment and nanofiltration]) in previously-treated patients aged 12 to 65 with severe (FIX level < 1%) or moderately severe (FIX level ≤ 2%) hemophilia B.
Methods:
Hemostatic efficacy after twice weekly prophylaxis with BAX326 was determined in terms of annualized bleeding rate (ABR) compared with a historical control group treated on- demand. PK equivalence was assessed between BAX326 and a commercial rFIX in a crossover design. Safety was evaluated by the occurrence of adverse events.
Summary:
In subjects on twice weekly prophylaxis with BAX326 over at least 3 months (N=56), 24 (43%) did not bleed throughout the study observation period, and the ABR was substantially lower when compared with a historical control group (79% reduction, p<0.001). Joint bleeds (major joints: wrist, elbow, shoulder, hip, knee, ankle) occurred at a mean ABR of 2.85 ± 4.25 compared with 1.41 ± 2.87 in non-joint bleed sites. Of the 32/56 subjects with bleeds, 90.6% (29/32) had arthropathy at screening and only 28.1% (9/32) did not have target joints, as compared to subjects without bleeds, of whom 79.2% (19/24) had arthropathy and 50% (12/24) did not have target joints at screening. Higher mean ABRs were observed in subjects with arthropathy (N=46) versus without arthropathy (N=8) (4.54 vs. 2.57 for all bleeds, 3.16 vs. 1.02 for joint bleeds, and 1.97 vs. 0.25 for spontaneous bleeds). A similar pattern was observed for the ABRs of joint bleeds and spontaneous bleeds in subjects with target joints (N=35) (mean ABR: 2.41 ± 3.79) and those with no target joints (N=21) (mean ABR: 0.58 ± 1.63). Most bleeds were controlled with 1-2 infusions of BAX326 and with an efficacy rating of “excellent.” BAX326 was equivalent to the comparator rFIX in terms of AUC 0 72 h /dose. BAX326 is safe and well tolerated in hemophilia B patients, with no signs of immunogenicity or thrombotic events.
Conclusions:
BAX326 has a positive safety profile and is efficacious in treating bleeds and in routine prophylaxis in PTPs aged ≥12 years with hemophilia B. The results also demonstrate that subjects with target joints and hemophilic arthropathy receiving secondary prophylaxis tend to have higher ABRs as compared to those without these underlying conditions.
This study explores perceptions among adolescent aged patients with inherited bleeding disorders regarding religiosity/spirituality and whether their beliefs have an impact on the pain they experience. Subjects participating will be assessed in regards to the type, frequency and intensity of pain experienced and measures used to cope with the pain. Finally, we will measure patient self reports of disease self efficacy. To further assess the impact of religiosity/spirituality on the family system, we will be measuring parental perceptions of religiosity/spirituality with parents of the participating adolescents.
