scholarly journals Prevention of the Musculoskeletal Complications of Hemophilia

2012 ◽  
Vol 2012 ◽  
pp. 1-7 ◽  
Author(s):  
E. C. Rodriguez-Merchan
Keyword(s):  
Prophylactic Treatment ◽  
Cost Effective ◽  
Venous Access ◽  
Joint Disease ◽  
Clotting Factor ◽  
Hemophilic Arthropathy ◽  
Chronic Synovitis ◽  
Clotting Factor Concentrates ◽  
Venous Access Devices

Hemophilia is an inherited disorder of clotting factor deficiencies resulting in musculoskeletal bleeding, including hemarthroses, leading to musculoskeletal complications. The articular problems of hemophiliac patients begin in infancy. These include: recurrent hemarthroses, chronic synovitis, flexion deformities, hypertrophy of the growth epiphyses, damage to the articular cartilage, and hemophilic arthropathy. The most commonly affected joints are the ankle, the knee, and the elbow. Hematologic prophylactic treatment from ages 2 to 18 years could avoid the development of hemophilic arthropathy if the concentration of the patient's deficient factor is prevented from falling below 1% of normal. Hemarthroses can be prevented by the administration of clotting factor concentrates (prophylaxis). However, high costs and the need for venous access devices in younger children continue to complicate recommendations for universal prophylaxis. Prevention of joint arthropathy needs to focus on prevention of hemarthroses through prophylaxis, identifying early joint disease through the optimal use of cost-effective imaging modalities and the validation of serological markers of joint arthropathy. Screening for effects on bone health and optimal management of pain to improve quality of life are, likewise, important issues. Major hemarthrosis and chronic hemophilic synovitis should be treated aggressively to prevent hemophilic arthropathy.

scholarly journals Haemophilia and Joint Disease: Pathophysiology, Evaluation, and Management

2011 ◽  
Vol 1 (1) ◽  
pp. 51-59 ◽  
Author(s):  
Karin Knobe ◽  
Erik Berntorp
Keyword(s):  
Joint Disease ◽  
Clotting Factor ◽  
Recurrent Bleeding ◽  
Effective Prevention ◽  
Haemophilic Arthropathy ◽  
Chronic Synovitis ◽  
Bleeding Episodes ◽  
End Stage ◽  
Clotting Factor Concentrates

In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles. However, despite this success, intra-articular and intramuscular bleeding is still a major clinical manifestation of the disease. Bleeding most commonly occurs in the knees, elbows, and ankles, and is often evident from early childhood. The pathogenesis of haemophilic arthropathy is multifactorial, with changes occurring in the synovium, bone, cartilage, and blood vessels. Recurrent joint bleeding causes synovial proliferation and inflammation (haemophilic synovitis) that contribute to end-stage degeneration (haemophilic arthropathy); with pain and limitation of motion severely affecting patients’ quality of life. If joint bleeding is not treated adequately, it tends to recur, resulting in a vicious cycle that must be broken to prevent the development of chronic synovitis and degenerative arthritis. Effective prevention and management of haemophilic arthropathy includes the use of early, aggressive prophylaxis with factor replacement therapies, as well as elective procedures, including restorative physical therapy, analgesia, aspiration, synovectomy, and orthopaedic surgery. Optimal treatment of patients with haemophilia requires a multidisciplinary team comprising a haematologist, physiotherapist, orthopaedic practitioner, rehabilitation physician, occupational therapist, psychologist, social workers, and nurses.

Infection of totally implantable venous access devices: A review of the literature

2018 ◽  
Vol 19 (3) ◽  
pp. 230-242 ◽  
Author(s):  
Fulvio Pinelli ◽  
Elena Cecero ◽  
Dario Degl’Innocenti ◽  
Valentina Selmi ◽  
Rosa Giua ◽  
...  
Keyword(s):  
Risk Factors ◽  
Venous Access ◽  
Outpatient Setting ◽  
Intravenous Therapy ◽  
Access Device ◽  
Venous Access Device ◽  
Treatment Of Infection ◽  
Venous Access Devices

Totally implantable venous access devices, or ports, are essential in the therapeutic management of patients who require long-term intermittent intravenous therapy. Totally implantable venous access devices guarantee safe infusion of chemotherapy, blood transfusion, parenteral nutrition, as well as repeated blood samples. Minimizing the need for frequent vascular access, totally implantable venous access devices also improve the patient’s quality of life. Nonetheless, totally implantable venous access devices are not free from complications. Among those, infection is the most relevant, affecting patients’ morbidity and mortality—both in the hospital or outpatient setting—and increasing healthcare costs. Knowledge of pathogenesis and risk factors of totally implantable venous access device–related infections is crucial to prevent this condition by adopting proper insertion bundles and maintenance bundles based on the best available evidence. Early diagnosis and prompt treatment of infection are of paramount importance. As a totally implantable venous access device–related infection occurs, device removal or a conservative approach should be chosen in treating this complication. For both prevention and therapy, antimicrobial lock is a major matter of controversy and a promising field for future clinical studies. This article reviews current evidences in terms of epidemiology, pathogenesis and risk factors, diagnosis, prevention, and treatment of totally implantable venous access device–related infections.

scholarly journals Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens

Blood ◽  
2015 ◽  
Vol 125 (13) ◽  
pp. 2038-2044 ◽  
Author(s):  
Johannes Oldenburg
Keyword(s):  
Treatment Strategies ◽  
Coagulation Factor ◽  
Pharmacokinetic Profile ◽  
Primary Prophylaxis ◽  
Secondary Prophylaxis ◽  
Joint Disease ◽  
System Level ◽  
Clotting Factor ◽  
Individualization Of Therapy ◽  
Clotting Factor Concentrates

Abstract Prophylactic application of clotting factor concentrates is the basis of modern treatment of severe hemophilia A. In children, the early start of prophylaxis as primary or secondary prophylaxis has become the gold standard in most countries with adequate resources. In adults, prophylaxis is reasonably continued when started as primary or secondary prophylaxis in childhood to maintain healthy joint function. Initial data support that adult patients with already existing advanced joint arthropathy benefit from tertiary prophylaxis with significantly lowered number of bleeds, almost complete absence of target joints, and less time off from work. Current prophylactic regimens, although very effective, do not completely prevent joint disease in a long-term perspective. Joint arthropathy in primary prophylaxis develops over many years, sometimes over a decade or even longer time periods. The ankle joints are the first and most severely affected joints in those patients and thus may serve in outcome assessment as an indicator of early joint arthropathy when followed by ultrasound or magnetic resonance imaging. Optimized outcome and best use of available resources is expected from individualization of therapy regimens, which comprises the individual’s bleeding pattern, condition of the musculoskeletal system, level of physical activity and the pharmacokinetic profile of the substituted coagulation factor, and most recently includes novel products with extended half-lives.

Radioisotope Synovectomy for Treating Chronic Synovitis in Inhibitor Patients with Hemophilia.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 4005-4005 ◽  
Author(s):  
Kaan Kavakli ◽  
Semih Aydogdu ◽  
Yusuf Duman ◽  
Memduh Taner ◽  
Guray Saydam ◽  
...  
Keyword(s):  
Hemophilia A ◽  
Cost Effective ◽  
Late Recurrence ◽  
Treatment Modalities ◽  
High Dose ◽  
Hemophilic Arthropathy ◽  
Chronic Synovitis ◽  
Grade Ii ◽  
Yttrium 90 ◽  
Grade Iii

Abstract Hemophilic arthropathy, caused by chronic synovitis is the most common musculoskeletal complication of recurrrent hemarthrosis in hemophilia patients. However, in patients with target joints and chronical synovitis, local directed therapy is more useful than systemic treatment. Radioisotope Synovectomy (RS) consists of destruction of synovial tissue by intra-articular injection of a radioisotope agent. RS has gained acceptance worldwide for 10 years after favourable reports in respect of efficacy and safety. The most frequent and serious complication in hemophilia-A is the development of inhibitors. Due to problems for hemostasis and limited treatment modalities, arthropathy risk is higher in these patients. For last 6 years, we have performed 163 RS procedures in 109 children and young adults in our center. In this report, we present our experience in inhibitor patients (16 cases and 28 joints). All cases were severe hemophilia-A (FVIII<2%). Thirteen patients had high responder (HR)(5–400 BU/ml), three had low responder (LR)(2–5 BU/ml) inhibitor. All patients had target joints and grade-II (n=8) and grade-III (n=20) synovitis. We have used intra-articularly 5 mCi Yttrium 90 for knees (n=16) and 2 mCi Yttrium 90 (n=7) or Rhenium 186 (n=5) for small joints (Schering-CIS, France). The age range was 3–26 years (mean 14.6±6.1yr). We have 4 patients below 10 years. The knees injected 16, elbows 7, ankles 3 and shoulders in 2 joints. Written informed consent was mandatory. For HR patients; recombinant FVIIa (Novoseven) was used (90 mcg/bw / in 2-h interval) in three consecutive doses except one case for 15 joints in 9 patients. Activated PCC (FEIBA) was used (75 IU/bw / in 12 h of interval) in 2 or 3 consecutive doses for 4 patients in 8 joints. High dose FVIII were used for 3 LR patients in 5 joints. Overnight hospitalization was applied. Mean follow-up period after procedure was 2.5 years (range: 6 month-5 year). The efficacy of procedure was evaluated by comparing joint bleedings and signs for synovitis. Seventy-five percent of inhibitor patients had satisfactory results. Pre-procedure joint bleeds were 7.9±3.2 (range: 4–16) for 6 months. After RS, during the first year hemarthroses were significantly decreased as (0.4±1.1). In cases with grade-II synovitis outcome was more better than grade-III synovitis as expected. In two patients, procedure was needed to be repeated due to insufficient effect. After repeated applications, outcome was satisfactory. In other two patients, late recurrence of synovitis was observed after 2 years. One of them, intra-articular injection was repeated and synovitis has improved. No complications have seen. Recently, some suspicions arose due to two children who had had the radioisotope synovectomy using with P32 subsequently developed acute leukemia (ALL) in the USA. However, we have used Y90 and Re186 and no malignancy have reported in the literature and not observed in our study group for 6 years. In conclusion, RS is efficient also in inhibitor patients as far as other patients. RS is cost-effective and easy to perform and hence highly recommended for inhibitor patients. RS seems to be invaluable for treatment of chronic synovitis of inhibitor patients for preventing hemophilic arthropathy.

Making the Case for a Nurse-Led Vascular Access Team Utilizing a Quality Assurance Conceptual Framework

2009 ◽  
Vol 14 (2) ◽  
pp. 77-82 ◽  
Author(s):  
Amanda L. Beerman
Keyword(s):  
Health Care ◽  
Health Care Providers ◽  
Financial Burden ◽  
Central Venous Access ◽  
Venous Access ◽  
Care Providers ◽  
Quality Of Patient Care ◽  
The Cost ◽  
Venous Access Devices

Abstract The cost of complications of central venous access devices (CVADs) in today's health care setting is creating a financial burden, increasing the expenditures of resources, and consuming the time of health care providers to resolve. As the roles of nurses continue to evolve, nurses may be in a position to recognize and prevent many of these complications. This paper proposes a conceptual model to address the issues related to caring for and monitoring CVADs, while reducing complications and increasing quality of patient care.

Successful Long-Term Prophylactic Treatment With Subcutaneous C1 Esterase Inhibitor in a Patient With Hereditary Angioedema

2019 ◽  
Vol 33 (6) ◽  
pp. 907-911
Author(s):  
Janina Hahn ◽  
Melanie Nordmann-Kleiner ◽  
Susanne Trainotti ◽  
Thomas K. Hoffmann ◽  
Jens Greve
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Prophylactic Treatment ◽  
Upper Airway ◽  
Venous Access ◽  
Current Standard ◽  
C1 Esterase Inhibitor ◽  
Esterase Inhibitor

Background: Hereditary angioedema (HAE) patients suffer from recurrent swellings. Current standard therapy consists of C1 esterase inhibitor (C1-INH) and bradykinin receptor B2 antagonists. Severe courses require prophylactic treatment. For such patients, it has been demonstrated that the intravenous (IV) administration of C1-INH [C1-INH(IV)] is safe and effective. A new prophylactic option is subcutaneous (SC) treatment with C1-INH. Methods and Case: We present the case of an HAE patient placed on prophylactic C1-INH(IV) therapy due to frequent attacks when managed with on-demand therapy. An implanted port allowed the periodical and safe application of medication until the device was explanted due to an infection. Due to the poor venous access, repeated IV application failed. Therefore, we began a SC treatment with 1500 IU C1-INH [C1-INH(SC)] as long-term prophylaxis and analyzed the clinical course over 16 months. Results: Under the SC prophylaxis, the number of attacks were reduced to 1/month in comparison to 4.33/month with no prophylactic treatment and 1.83/month with C1-INH(IV). No severe attacks and no attack within the upper airway occurred over the 16 months of C1-INH(SC) treatment. As a result, quality of life improved, as measured by the Angioedema quality of life questionaire (AE-QoL). Conclusion: Self-administered SC prophylactic use of C1-INH over a period of 16 months seems to be a well tolerated and efficient. The patient’s quality of life improved, and by learning self-application, the patient gained independence.

scholarly journals How I use bypassing therapy for prophylaxis in patients with hemophilia A and inhibitors

Blood ◽  
2015 ◽  
Vol 126 (2) ◽  
pp. 153-159 ◽  
Author(s):  
Cindy A. Leissinger ◽  
Tammuella Singleton ◽  
Rebecca Kruse-Jarres
Keyword(s):  
Physical Disability ◽  
Hemophilia A ◽  
Joint Disease ◽  
Clotting Factor ◽  
On Demand ◽  
Increased Risk ◽  
Related Quality ◽  
Health Related ◽  
Bleeding Episodes

Abstract Inhibitor development poses a significant challenge in the management of hemophilia because once an inhibitor is present, bleeding episodes can no longer be treated with standard clotting factor replacement therapy. Consequently, patients with inhibitors are at increased risk for difficult-to-control bleeding and complications, particularly arthropathy and physical disability. Three clinical trials in patients with inhibitors have demonstrated that prophylaxis with a bypassing agent reduces joint and other types of bleeding and improves health-related quality of life compared with on-demand bypassing therapy. In hemophilia patients without inhibitors, the initiation of prophylaxis with factor (F) VIII or FIX prior to the onset of recurrent hemarthroses can prevent the development of joint disease. Whether this is also true for bypassing agent prophylaxis remains to be determined.

The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia

Blood ◽  
2002 ◽  
Vol 99 (7) ◽  
pp. 2337-2341 ◽  
Author(s):  
Kathelijn Fischer ◽  
Johanna G. van der Bom ◽  
Eveline P. Mauser-Bunschoten ◽  
Goris Roosendaal ◽  
Robert Prejs ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Joint Damage ◽  
Patient Characteristics ◽  
Clotting Factor ◽  
Severe Hemophilia ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Hemophilic Arthropathy

To prevent hemophilic arthropathy, prophylactic treatment of children with severe hemophilia should be started before joint damage has occurred. However, treatment is expensive, and the burden of regular venipunctures in young children is high. With the aim of providing information on starting prophylaxis on the basis of individual patient characteristics, the effect of postponing prophylaxis on long-term arthropathy was studied in a cohort of 76 patients with severe hemophilia born between 1965 and 1985. The median age at first joint bleed was 2.2 years (range, 0.2-5.8). Prophylaxis was started at a median age of 6 years (interquartile range [IQR], 4-9), and the median annual clotting factor use on prophylaxis was 1750 IU/kg/y (31 IU/kg/wk). Hemophilic arthropathy was measured by the Pettersson score (maximum, 78 points). At a median age of 19 years, the median Pettersson score was 7 points (IQR, 0-17). After 2 decades of follow-up, the Pettersson score was 8% higher (95% confidence interval, 1%-16%) for every year prophylaxis was postponed after the first joint bleed. This effect was independent of age at Pettersson score, age at first joint bleed, and prophylactic dose used. In conclusion, most patients have their first joint bleed after the age of 2 years. Patients who start prophylaxis soon after the first joint bleed show little arthropathy in adulthood. The longer the start of prophylaxis is postponed after the first joint bleed, the higher the risk of developing arthropathy.

Safe and Cost Effective Use of Alteplase for the Clearance of Occluded Central Venous Access Devices

2002 ◽  
Vol 20 (7) ◽  
pp. 1918-1922 ◽  
Author(s):  
J. P. Timoney ◽  
M. G. Malkin ◽  
D. M. Leone ◽  
J. S. Groeger ◽  
M. L. Heaney ◽  
...  
Keyword(s):  
Thrombolytic Agent ◽  
Central Venous Access ◽  
Cost Effective ◽  
Venous Access ◽  
Central Venous ◽  
Safety And Efficacy ◽  
Reasonable Alternative ◽  
Effective Use ◽  
Assessment Initiative ◽  
Venous Access Devices

PURPOSE: To determine whether cryopreserved solutions of the thrombolytic agent alteplase could be used as a safe, effective, and economically reasonable alternative to urokinase in patients presenting with occluded central venous access devices (CVADs). MATERIALS AND METHODS: Alteplase has been reported as an efficacious alternative to urokinase for treatment of occluded CVADs. However, the practicality of using alteplase as the thrombolytic of choice for this indication remained conjectural. To make this approach economically feasible, alteplase was diluted to 1 mg/mL and 2.5-mL aliquots were stored at −20°C until use. A need to confirm that the cryopreserving and thawing of the reconstituted solution did not compromise the safety and efficacy reported from prior trials was recognized. A quality assessment initiative was undertaken to concurrently monitor the safety and efficacy of this approach. Patients presenting with occluded CVADs received a sufficient volume of the thawed alteplase solution to fill the occluded catheter(s). Data, including efficacy, adverse reactions, dwell time, and catheter type, were collected over a 5-month period. RESULTS: One hundred twenty-one patients accounting for 168 attempted clearances were assessable for safety and efficacy. One hundred thirty-six (81%) of the 168 catheter clearance attempts resulted in successful catheter clearance (95% confidence interval, 74% to 86%). No adverse events were reported. CONCLUSION: Cryopreserved 1-mg/mL aliquots of alteplase are safe and effective in the clearance of occluded CVADs when stored at −20°C for 30 days. The ability to cryopreserve alteplase aliquots makes it an economically reasonable alternative to urokinase in the setting of CVAD occlusion.

scholarly journals How do we optimally utilize factor concentrates in persons with hemophilia?

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 206-214
Author(s):  
Ming Y. Lim
Keyword(s):  
Standard Of Care ◽  
Clotting Factor ◽  
World Federation ◽  
Musculoskeletal Health ◽  
Related Factors ◽  
Tailored Approach ◽  
Individualized Management ◽  
Clotting Factor Concentrates ◽  
Factor Concentrate

Abstract The current mainstay of therapy for hemophilia is to replace the deficient clotting factor with the intravenous administration of exogenous clotting factor concentrates. Prophylaxis factor replacement therapy is now considered the standard of care in both pediatric and adult patients with hemophilia with a severe phenotype to protect musculoskeletal health and improve quality of life. Heterogeneity in bleeding presentation among patients with hemophilia due to genetic, environmental, and treatment-related factors has been well described. Accordingly, the World Federation of Hemophilia recommends an individualized prophylaxis regimen that considers the factors mentioned above to meet the clinical needs of the patient, which can vary over time. This review focuses on the practical points of choosing the type of factor concentrate, dose, and interval while evaluating appropriate target trough factor levels and bleeding triggers such as level of physical activity and joint status. We also discuss the use of a pharmacokinetics assessment and its incorporation in the clinic for a tailored approach toward individualized management. Overall, adopting an individualized prophylaxis regimen leads to an optimal utilization of factor concentrates with maximum efficacy and minimum waste.

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