Objective:
Using point-of-care musculoskeletal ultrasound (MSKUS), we previously demonstrated that patient and physician assessments were unreliable in determining bleeding during acute painful joint episodes. Here we delineated by MSKUS pathophysiological soft tissue changes that may contribute to pain, and investigated to what extent MSKUS findings and functional or radiographic joint status correlate with markers of inflammation.
Methods:
We used the GE Logiq e BT11 US-module with high frequency 8-13 MHz linear transducer and real time spatial compound imaging capability for grey scale and Power Doppler examinations. We analyzed all MSKUS examinations performed between 05/2012 and 08/2013 in 34 adult hemophilia patients (mean age 39.3 years) seen at our Hemophilia Treatment Center. Findings were correlated with Hemophilia Joint Health Scores (HJHS), Pettersson Scores, hsCRP, and von Willebrand Factor (VWF) activity and antigen levels. Spearman correlation coefficient and Wilcoxon Mann-Whitney tests were used. P-values ≤0.05 were considered significant. Acute and persistent pain was defined as lasting ≤7 days and >7 days, respectively.
Results:
Sixty-five examinations were performed. Seventy percent of patients had severe hemophilia. Mean Pettersson scores were 22 of 78 and HJHS were 22 of 124. Joints most commonly examined were knees and ankles (72%), with most examinations (72%) performed for persistent pain. Effusions were present in 48% of painful joints. Of those effusions, 90% were bloody during acute and ~50% during persistent pain episodes. Synovitis (+/-tendinitis, enthesitis or bursitis) was observed in 66% of all MSKUS examinations. Synovitis and hemarthrosis coincided in 20% of examinations. In exams revealing hemarthrosis, synovitis was present in 68%. In acute hemarthrosis, synovitis was present in 55% and, with persistent pain, synovitis was present in 80%. Although total and joint-specific HJHS and Pettersson scores were higher in patients with synovitis, only the joint-specific Pettersson score was significantly higher (mean score 3 vs 6.5). HsCRP, VWF activity and VWF antigen levels correlated significantly with joint-specific Pettersson scores (Cr ~0.4) and total HJHS (Cr ~0.6), but not consistently with synovitis.
Conclusion:
Inflammation and bleeding were prominent findings in painful hemophilic arthropathy. One-fifth of persistently painful joints were diagnosed with hemarthroses, which were almost always associated with synovitis. Inflammatory markers correlated to some extent with joint findings, but were diagnostically not helpful. We conclude that sensitive imaging technology such as MSKUS is critical to precisely diagnose causes of pain in hemophilic arthropathy with a need for personalized care that includes tailored clotting factor replacement and/or novel anti-inflammatory strategies.
As technology advances and provides electronic tools for enhancing communication by phone and computer, health care providers are finding ways to adapt these tools into patient care. Telehealth is the use of electronic information and telecommunications technologies to support long-distance clinical health care. For patients with hemophilia who experience a bleed in the home setting, telehealth has the potential to help the patient, family, and health care provider assess what is going on and develop the best plan of care, all while the patient stays in the home setting. In this clinical project, we will use the telehealth resources available at our institution to partner with patients and families with severe hemophilia with a high risk of bleeding episode who also have a home computer with a camera and internet access. We want to find out more about how many patients have these home resources, how to use video-conferencing when managing a bleed and what patients, families, and health care staff think about using video conferencing. This will help us plan future research using telehealth video-conferencing for a larger group of hemophilia patients.
Background/Aim:
Little data exist, especially for adolescents and young adults (AYAs), about the relationship between adherence to prescribed hemophilia treatment regimens and chronic pain (CP).
Methods:
A convenience sample of hemophiliacs aged 13-25 completed an IRB-approved, online survey addressing regimen-specific adherence and CP between April through December of 2012. Adherence was assessed for prophylactic (VERITAS-Pro) and on- demand (VERITAS-PRN) participants. VERITAS scores range from 24 (most adherent) to 120 (least adherent). CP was measured using the revised Faces Pain Scale (FPS-R). CP was dichotomized as high (‘moderate’ to ‘worst pain possible,’ i.e., ≥4) or low (‘mild’ or ‘no pain,’ (i.e., <4). Multivariable, parsimonious logistic regression models assessed factors associated with high vs low CP levels. Separate models were constructed to evaluate a combined VERITAS score among prophylactic and on-demand patients and the VERITAS- Pro score among prophylactic patients only. Small sample size precluded analysis of on- demand (only) participants.
Results:
Ninety-three AYAs participated. Mild patients (n=13) were excluded. Of the remaining 80 participants (79 male), 91% had severe disease, 86% infused prophylactically, and 91% had Hemophilia A. Fifty-one percent were aged 13-17, most were white (76%), non- Hispanic (88%), and never married (93%). The majority (94%) had some type of health insurance.
Mean VERITAS-Pro (n=69) and PRN (n=11) scores were 49.6 ±12.9 (range 25-78) and 51.0 ±11.6 (range 35-74), respectively. CP was reported as high for 35% of respondents (36% for prophylactic vs 27% for on-demand, p=.74). Mean VERITAS-Pro scores for those with high and low CP were 53.6 ±12.3 vs 47.4 ±12.9, p=.05. VERITAS-PRN scores were similar across CP status. Logistic regression analysis revealed that for each 10-point reduction (increase in adherence) in the combined VERITAS score (Pro and PRN) there was a 35% (OR=0.65; 95%CI=0.44, 0.96; p=.03) reduction in the odds of having high CP. Among prophylactic respondents: for each 10-point reduction in the VERITAS-Pro score there was a 39% (OR=0.61; 95%CI=0.39, 0.96; p=.03) reduction in the odds of having high CP and compared to whites, non-whites were 4.42 (95%CI: 1.21, 16.1; p=.02) times as likely to report high CP.
The goal of Dr. Chen's research is to examine a method for selectively expanding hematopoietic stem cells expressing the factor VIII transgene. She will also examine the immune consequences of this approach, based on the idea that gene transfer in platelets evades immune recognition. This research has the potential to elicit important clues to developing an approach for gene therapy of hemophilia A and hemophilia A with inhibitors.
Dr. Chen earned a PhD in hematology from Fujian Medical University in China. She already has more than 27 papers published in the Chinese medical literature. Her research in hemophilia and gene therapy will be under the mentorship of Dr. Qizhen Shi, MD, PhD, Associate Investigator at the Blood Research Institute and Assistant Professor of Pediatric Hematology at the Medical College of Wisconsin.
Objective:
To assess subjective and objective outcomes of total joint arthroplasty (TJA) as a treatment for hemophilic arthropathy, and to assess the safety and efficacy of perioperative pharmacologic thromboprophylaxis as a means to prevent venous thromboembolism in this population.
Methods:
We performed a retrospective chart review to identify patients with congenital bleeding disorders who underwent TJA between 1987 and 2012. We collected data on range of motion (ROM) and pain before and after surgery and on early and late complications (bleeding, infection, thrombosis). Data are presented descriptively using median values and ranges where appropriate.
Summary:
We identified 38 procedures (29 knees (TKA) and 9 hips (THA) in 28 patients (26 male, 2 female) with hemophilia A (n = 21), hemophilia B (n = 4), factor 11 deficiency (n = 1) and von Willebrand disease (n = 2). Median age at operation was 42 years (range, 17 – 74) for TKA and 45 years (range, 18 – 71) for THA. Inhibitors were present in one patient with hemophilia A (1.5 B.U.) and one patient with factor 11 deficiency (0.5 B.U.). All patients were treated with hemostatic agents appropriate to their disorders for up to 4 to 6 weeks post- operatively. Complete data at 2 months post-operatively are available for 27 TKA patients, of whom, 7 (23%), demonstrated improvement in ROM (median 15 degrees, range 5 - 25). At 1.5 years post-operatively, 17/29 (59%) TKA patients showed improvement in ROM (median 15 degrees, range 4 - 58) and 100% reported decreased knee pain. All 9 THA patients demonstrated improved ROM at 2 months post-operatively. Eight (89%) demonstrated gains in internal rotation (median, 45 degrees, range 15 – 45), 9 (100%) in external rotation (median 30 degrees, range 15 – 45), 5 (56%) in flexion (median 35 degrees, range 27 – 55), 7 (78%) in extension (median 15 degrees, range 3 – 95), and 7 (78%) in abduction (median 15 degrees, range 10 – 25).
We were able to contact 22 of 28 study subjects (79%), accounting for 31 of 38 (82%) procedures. Patients who underwent 25 of the 29 TKAs (86%) and 6 of the 9 THAs (67%) agreed to provide answers to yes/no questions about their experience with TJA. 25 of 25 (100%) TKA subjects reported improvement in pain and stated that if given the opportunity to go back and revisit their decision, would make the same decision to have the surgery. 24 of 25 (96%) TKA subjects reported improvement in their joint function after the surgery. 6 of the 6 THA subjects we contacted stated that they experienced improvement in joint pain and function as a result of the surgery, and 5 of 6 (83%) stated that they would choose to have the surgery if they had to choose again.
Low molecular weight heparin was administered post-operatively in 29 of 38 procedures (76%). Thromboprophylaxis was discontinued in 3 patients for non-joint bleeding (one hematuria, two cases of hypotension and anemia). There were no symptomatic VTE. Early complications included 5 cases of cellulitis and 2 hemarthroses in patients not receiving thromboprophylaxis. Late complications included two patients with aseptic loosening in prosthetic knees leading to TKA revisions, one with a subsequent joint infection requiring surgical debridement and one patient with a worsening flexion contracture requiring TKA revision.
Conclusions:
While there are risks associated with TJA in patients with bleeding disorders, our data suggest they are outweighed by the benefits manifesting as decreased pain and improved function. Pharmacologic thromboprophylaxis appears safe in this population; whether it is necessary is unknown and should be a subject of future trials.
