scholarly journals Effect of tertiary prophylaxis with low-dose factor VIII in quality of life in adult patients with severe hemophilia A

2019 ◽  
Vol 10 (3) ◽  
pp. 88
Author(s):  
PrakasKumar Mandal ◽  
Abhijit Phukan ◽  
Amrita Bhowmik ◽  
Debasis Gantait ◽  
Prantar Chakrabarti
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Low Dose ◽  
Hemophilia A ◽  
Adult Patients ◽  
Severe Hemophilia ◽  
Dose Factor

scholarly journals Low dose long-acting factor VIII prophylaxis in pediatric and young adult patients with hemophilia A: Short-term single-center experience from a developing country

2021 ◽  
Vol 0 ◽  
pp. 1-6
Author(s):  
Shailendra Prasad Verma ◽  
Anil Kumar Tripathi ◽  
Geeta Suri Sharma ◽  
Nidhish Kumar ◽  
Rashmi Kushwaha
Keyword(s):  
Young Adult ◽  
Adverse Events ◽  
Factor Viii ◽  
Low Dose ◽  
Hemophilia A ◽  
Adult Patients ◽  
Severe Hemophilia ◽  
Emergency Visits ◽  
Episodic Treatment ◽  
Long Acting

Objectives: High dose factor prophylaxis in hemophilia has been proven to prevent joint bleeds in the western world effectively. We look for a cost-effective and feasible way for Indian patients to reduce the dose and frequency of factor infusion. Data on prophylaxis with a low dose, long-acting factor infusion twice a week dosing schedule is limited. The purpose was to study the efficacy and safety of long-acting factor VIII (Eloctate) for secondary/ tertiary prophylaxis in pediatric and young adult patients with moderate and severe hemophilia A. Materials and Methods: Thirty-eight patients with moderate and severe hemophilia A with an age range from 1 to 25 years were included in the study. During the initial 4 months, they received therapeutic doses of ELOCTATE (Factor VIII with Fc Fusion Protein) on an episodic basis after a clinical bleed. In the next 4 months, they received prophylactic intravenous ELOCTATE at the dose of 20 units/kg body weight twice a week. Annual bleeding rates (ABR), school absenteeism, emergency visits, joint scores, and adverse events were compared during both periods. Results: The total number of joint bleeds during the episodic treatment and prophylaxis period was 608 and 67, respectively. ABR was 47.9 during the episodic treatment period and 5.3 during prophylaxis showing an 88.9% reduction in joint bleeds. School/college absenteeism and emergency visits were significantly reduced during prophylaxis. No significant adverse events were noted during prophylaxis. Conclusion: Low dose, twice a week, and long-acting recombinant factor VIII-Fc (Eloctate) prophylaxis can be a reasonable options for patients with hemophilia A in developing countries.

Factor VIII Intron 22 Inversion Screening of Newborn Males for Hemophilia A: A Cost-Effectiveness Study

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 734-734
Author(s):  
John W. Luiza ◽  
Margaret V. Ragni ◽  
Robert F. Sidonio ◽  
Kenneth J Smith
Keyword(s):  
Quality Of Life ◽  
Cost Effectiveness ◽  
Factor Viii ◽  
Hemophilia A ◽  
Severe Hemophilia ◽  
Pcr Screening ◽  
Intron 22 Inversion ◽  
Screening Cost ◽  
The Cost

Abstract Abstract 734 Background: Severe hemophilia A is an X-linked congenital bleeding disorder occurring in 1:5,000 male births. Among neonates with severe hemophilia A, failure to recognize hemophilia and associated bleeding may result in severe blood loss anemia from circumcision, central nervous system (CNS) bleeding during the birth process or with head trauma and associated neurologic sequelae, and unrecognized joint bleeding that, when recurrent, increases the risk of joint damage which may lead to chronic disability. In at least one-third of cases, the disease arises as a spontaneous mutation: yet, even among the two-thirds with a family history, most carriers do not undergo carrier testing or prenatal diagnosis, leaving only a minority in whom cord blood screening is performed. About half of newborns with severe hemophilia A have a factor VIII (F.VIII) intron 22 inversion mutation, readily detected by PCR screening. We, therefore, sought to determine the effects of newborn screening by F.VIII intron 22 inversion PCR on early diagnosis in children with severe hemophilia A, specifically, on prevention of early life bleeding and associated cost, morbidity, and quality of life. Methods: We constructed a decision tree model to evaluate the cost effectiveness of newborn F.VIII intron 22 screening for severe hemophilia A. We assumed all newborn males were tested as part of screening, and that treatment modifies the likelihood of bleeding but not bleeding associated morbidity. Rates of major and minor CNS, joint, and procedural/surgical bleeding, including circumcision, morbidity and mortality, cost, and quality of life utilities were obtained from the literature. We assumed the cost of intron 22 PCR testing to be $3.00 per newborn male, that test results were available within 2 days of screening, and that clotting factor was infused prior to procedures and at the first sign of joint bleeding or head trauma. The probability of severe bleeding requiring hospitalization or red blood cell transfusion was estimated to be 5% or less in children with severe hemophilia A. The cost of F.VIII concentrate was based on the average wholesale price, and transfusion and hospitalization costs were based on local data. Outcomes included medical costs for each bleeding event, effectiveness measured as quality-adjusted-life-years (QALY), and the incremental cost-effectiveness ratio (ICER) over the first two years of life. Sensitivity analysis was used to test the robustness of analysis results. Results: Compared to no screening, screening for hemophilia had an ICER of $96,918/QALY, a value considered economically reasonable. Results were sensitive to variation of screening cost and overall detection of hemophilia A by PCR screening (base case 50%). Effects of varying both these parameters in a two-way sensitivity analysis are shown in the Figure. Using a $100,000 per QALY cost-effectiveness criterion over the depicted ranges for both parameters, screening was favored if screening cost ≤$3 or if ≥56% of all newborns with hemophilia A were detected by screening. Conclusion: It is cost effective to perform factor VIII intron 22 PCR screening to identify severe hemophilia A in newborn males in order to prevent bleeding morbidity, if the cost of the test does not exceed $3.00. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Health status and quality of life in patients with severe hemophilia A: A cross-sectional survey

2019 ◽  
Vol 11 (2) ◽  
Author(s):  
Majid Davari ◽  
Zahra Gharibnaseri ◽  
Roya Ravanbod ◽  
Abolfazl Sadeghi
Keyword(s):  
Quality Of Life ◽  
Hemophilia A ◽  
Clinical Information ◽  
Cross Sectional Study ◽  
Adult Patients ◽  
Severe Hemophilia ◽  
Cross Sectional Survey ◽  
Cross Sectional ◽  
The Mean

Among different groups of hemophiliacs, those suffering from Severe Hemophilia A (SHA) are most vulnerable to the complications of the disease. This study investigated the Health-Related Quality of Life (HR-QoL) among adult patients with SHA. A cross-sectional study was designed to gather demographic and clinical information from adult patients with SHA. Patients with inhibitors were excluded. The remaining were asked to complete the HR-QoL questionnaire after being examined for joint health using the Hemophilia Joint HealthScore (HJHS). The HR-QoL and joint conditions were measured in 38 patients. The mean EQ-5D value scores were 0.46 (SD=0.23) while the mean Visual Analogous Scale score was 50 (SD=18.7). The clinical examination of patients indicated that the HJHS were as follows: eight patients had a score of 55-75, 12 patients had a score of 40-55, 7 of them (25-40) and 11 patients had a score of 10-25. The results obtained from this study showed that HR-QoL in hemophilia patients was considerably low. Pain, anxiety/depression, and motion limitations were the main causes of the disutility for these patients respectively.

scholarly journals Comparison of Short-Term Tertiary Prophylaxis at Low-Dose and Intermediate-Dose for Adults with Severe Hemophilia a in China

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4675-4675
Author(s):  
Shunhua Huang ◽  
Zhitao Li ◽  
Yang Liu ◽  
Fangmei Qin ◽  
Xiaoqin Feng ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Dose Regimen ◽  
Dose Group ◽  
Low Dose ◽  
Hemophilia A ◽  
P Value ◽  
Severe Hemophilia ◽  
Dosing Intervals ◽  
Intermediate Dose

Abstract Background: Low-dose prophylaxis and intermediate-dose prophylaxis in hemophilia A both had been proved in reducing bleedings in some developing country, but there was rare attention in terms of the Health-related Quality of life (HRQoL) and patients' adherence. So we initiated a retrospective study to compare the differecen between low-dose and intermediate-dose regimen of tertiary prophylaxis for adults with severe hemophilia A. Methods: Data collected from the hemophilia treatment centre at Nanfang hospital from July 2010 to February 2015(median 2 years of follow-up), a total of 40 adult patients with severe hemophilia A (FVIII < 1%) who are receiving prophylaxis treatment≥1 year and treatment data available were eligible for enrollment in the study, including 25 patients in low-dose regimen(F‡[8-15 IU/kg 1-2times weekly or weekly<30IU/kg), and 15 patients in intermediate-dose regimen( F‡[ 15-20 IU/kg 2-3 times weekly or weekly≥40IU/kg and <75 IU/kg). Collect the data with patients' characteristics, previous condition and treatment, annual total bleeding rate(ABR). Use SF36 to evaluate patients' quality of life, use the difference between the beginning of this study and one year ago of SF36 score to represent the improvement of quality of life. Use "hemophilia bleeding and factor injection record handbook" to collect information about patients' bleeding and injection record, use HO's measuring method to evaluate adherence. Results: 1. There were no statistical differences between two groups in age, weight, age at first bleeding, age at first treatment, family history of hemophilia and history of hepatitis.(the range of P-value was 0.221-1.000). 2.The Health-related Quality of life (HRQoL) in patients with severe hemophilia A were measured by the SF-36. Only on bodily pain(P=0.965) and vitality(P=0.101)the differences did not exist between two groups ,beyond that intermediate-dose group were better in other aspects(included total SF36, physical functioning, role-physical, general health, social functioning, role-emotional and mental health) than low-dose group(the range of P-value was 0.000-0.045, P<0.05),which inferred that the improvement of quality of life was better in patients in intermediate-dose group. 3. Used HO's measuring method for adherence to evaluate the 40 patients in two groups. The dosage and dosing intervals adherence of low-dose group were 78.6A88.5 respectively, while the intermediate-dose group were 90.3A95.2 respectively, the differences in dosage and dosing intervals adherence between two groups were statistically significant(P=0.003). Intermediate-dose prophylaxis treatment reduced the annual total bleeding rate(ABR) than the low-dose group(median 13 vs. 5.5,P=0.000), while poor dosage adherence correlates with more ABR(B=-0.555,P=0.000), which meant worse adherence was association with much bleeding, but there was no correlation between dosing intervals adherence and ABR(B=0.171,P=0.507). Conclusion: 1. Compared to low-dose regimen, intermediate-dose regimen for short-term tertiary prophylaxis in adults with severe hemophilia A would improve quality of life. 2.Patients in intermediate-dose prophylaxis regimen had better adherence than low-dose regimen. There are negative correlation between dosage adherence and ABR, which means poor adherence lead to more bleeding events, while there's no correlation between dosing intervals adherence and ABR. Disclosures No relevant conflicts of interest to declare.

Health-related quality of life in a cohort of adult patients with mild hemophilia A

2008 ◽  
Vol 6 (5) ◽  
pp. 755-761 ◽  
Author(s):  
M. WALSH ◽  
D. MACGREGOR ◽  
S. STUCKLESS ◽  
B. BARRETT ◽  
M. KAWAJA ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Hemophilia A ◽  
Adult Patients ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related

scholarly journals Problem Joints and Their Clinical and Humanistic Burden in Children and Adults with Moderate and Severe Hemophilia a: CHESS Paediatrics and CHESS II

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 33-34
Author(s):  
Paul McLaughlin ◽  
Cedric Hermans ◽  
Sohaib Asghar ◽  
Tom Burke ◽  
Francis Nissen ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Research Funding ◽  
Hemophilia A ◽  
Joint Damage ◽  
Severe Hemophilia ◽  
Publicly Traded ◽  
Hemophilic Arthropathy ◽  
Joint Health ◽  
Humanistic Burden

Introduction Severe hemophilia A (SHA) is characterized by spontaneous (non-trauma related) bleeding episodes into the joint space and muscle tissue, leading to progressive joint deterioration and chronic pain. Chronic joint damage is most often associated with severe hemophilia, however more recent research has illustrated that people with moderate hemophilia A (MHA) also experience hemophilic arthropathy and functional impairment. The need to measure joint health in children as well as adults, is underscored by findings from the Joint Outcome Continuation Study, which found that FVIII prophylaxis was insufficient to protect joints from damage, from childhood through adolescence in severe HA (Warren et al., 2020). The objective of this analysis is to gain a more patient-centric understanding of the clinical, economic and humanistic burden associated with 'Problem Joints', a measure of joint morbidity developed in consultation with an expert panel to overcome limitations with existing measures, in people with MHA and SHA. Methods A descriptive cohort analysis was conducted, utilizing retrospective, cross-sectional real-world data from the 'Cost of Haemophilia in Europe: a Socioeconomic Survey' (CHESS Paeds and CHESS II), studies of adult and pediatric persons with hemophilia. The analysis population is comprised of children (17 and below) with MHA or SHA in CHESS Paeds, and adults aged 20 and over with MHA or SHA in CHESS II. To account for the possibility that persons aged 18 or 19 in CHESS II may have participated in CHESS Paeds, these individuals were excluded from the analysis. Physician-reported clinical outcome data and patient/caregiver-reported quality of life were analyzed. A problem joint (PJ) is defined as having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e. chronic synovitis and/or hemophilic arthropathy). Analyses were stratified by number of PJs: none, 1 PJ, and 2+ PJs. We report retrospective data of the 12 months prior to study enrollment, on annualized bleeding rate (ABR), prevalence of target joints (TJ), as defined by the International Society on Thrombosis and Haemostasis, and EQ-5D-/5L/Y/Proxy score. Results are presented as mean (standard deviation) or N (%). Results Among 785 participants (N = 464 SHA; N = 321 MHA) in CHESS Paeds, mean age and BMI were 10.33 (4.63) and 22.50 (17.07), respectively. Of 493 participants (aged 20 and above) in CHESS II (N = 298 SHA; N = 195 MHA), the mean age and BMI were 38.61 (14.06) and 24.55 (2.92), respectively. Current inhibitor to FVIII replacement was more prevalent in children than in adults (10% vs. 5%). In CHESS II, approximately 40% of people with MHA and 49% with SHA had one or more PJs, respectively [1 PJ (23% vs. 28%); 2+ PJs (16% vs. 21%)]. In CHESS Paeds, approximately 14% of children with MHA and 18% with SHA had at least one PJ, respectively [1 PJ (9% vs. 14%); 2+ PJs (5% vs. 3%)]. TJs were less prevalent with MHA in comparison to SHA, in both adults (24% vs. 45%) and children (13% vs. 22%). Clinical burden was higher among both children and adults with PJs compared to those with no PJs. ABR correlates with the number of PJs, in those with MHA and SHA in CHESS II (Figure 1). Similarly, PJs were associated with higher ABR across MHA and SHA in CHESS Paeds (Figure 2). Hemophilia-related hospitalizations were higher in both adult and pediatric participants with PJs. In CHESS II, MHA with no PJs had fewer [0.73 (1.23)] hospitalizations compared to having those with 1 PJ [1.38 (1.11)] or 2+ PJs [1.28 (1.25)]. Similarly, children with MHA with 2+ PJs had 1.60 (1.92) hemophilia-related hospitalizations, compared to 1.38 (1.92) with 1 PJ and 0.71 (1.14) with no PJs. PJs were associated with impaired quality of life. In CHESS II, MHA and SHA EQ-5D-5L values in persons with no PJs were 0.81 (0.19) and 0.79 (0.18), respectively, compared to 0.65 (0.16) and 0.62 (0.23) with 1 PJ, and 0.65 (0.14) and 0.51 (0.33) in with 2+ PJs. A similar trend was observed in EQ-5D-Y and EQ-5D-proxy scores in CHESS Paeds. Conclusions Data from CHESS Paeds and CHESS II demonstrate an association between chronic joint damage, as measured by the 'problem joint' definition, and worsening clinical and quality of life outcomes, across both MHA and SHA. Further analyses will seek to expand upon the initial results presented here, to investigate the wider elements of burden associated with compromised long-term joint health. Disclosures McLaughlin: BioMarin: Consultancy; Novo Nordisk: Consultancy, Speakers Bureau; Sobi: Consultancy, Speakers Bureau; Roche/Chugai: Speakers Bureau; Takeda: Speakers Bureau. Hermans:Novo Nordisk: Consultancy, Speakers Bureau; Roche: Consultancy, Speakers Bureau; Sobi: Consultancy, Research Funding, Speakers Bureau; Biogen: Consultancy, Speakers Bureau; CAF-DCF: Consultancy, Speakers Bureau; CSL Behring: Consultancy, Speakers Bureau; Shire, a Takeda company: Consultancy, Research Funding, Speakers Bureau; Pfizer: Consultancy, Research Funding, Speakers Bureau; Bayer: Consultancy, Research Funding, Speakers Bureau; WFH: Other; EAHAD: Other; Octapharma: Consultancy, Speakers Bureau; Kedrion: Speakers Bureau; LFB: Consultancy, Speakers Bureau. Asghar:HCD Economics: Current Employment. Burke:HCD Economics: Current Employment; University of Chester: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy. Nissen:GSK: Research Funding; Novartis: Research Funding; Actelion: Consultancy; F. Hoffmann-La Roche Ltd: Current Employment. Aizenas:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company. Meier:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company. Dhillon:HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. . O'Hara:F. Hoffmann-La Roche Ltd: Consultancy; HCD Economics: Current Employment, Current equity holder in private company.

Immune Tolerance Resulting in a Dramatic Clinical Improvement in a High-Responding Inhibitor Hemophilia A Patient Using Immunosuppression with Mycofenolate Mofetil and a Factor VIII Concentrate Containing Von Willebrand Factor.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3220-3220
Author(s):  
Thierry Lambert ◽  
Cécile Goujard ◽  
Anne Rafowicz ◽  
Benoît Guillet ◽  
Yacine Taoufik ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Clinical Improvement ◽  
Von Willebrand Factor ◽  
Immune Tolerance ◽  
Hemophilia A ◽  
Inhibitor Level ◽  
Von Willebrand ◽  
Willebrand Factor

Abstract A 40 year-old Caucasian patient with severe familial hemophilia A (FVIII <1%) related to an intron 22 inversion developed at the age of 2 a high-responding inhibitor. His elder brother also suffers from hemophilia A with high-responding inhibitor. Until 2000, the patient had been treated either using activated prothrombin complex concentrates (Autoplex® and Feiba®) or factor VIII (FVIII) concentrates of human (1983) and porcine origin (1992), resulting respectively in an inhibitor level rise at 360 Bethesda Units (BU) and 1800 BU. Between 2000 and 2003, the patient received exclusively recombinant activated Factor VII (NovoSeven®), and his inhibitor levels stabilized at a plateau of 15–20 BU. Between 2000 and 2003, several life or function threatening bleeding episodes occurred, such as hematomas of the iliopsoas muscles, spinal cord hematoma with transient paraplegia. Furthermore, due to hemarthroses the patient was confined to a wheelchair. Given the major impact of the inhibitor on the patient’s functional prognosis, life expectancy and quality of life, immune tolerance (IT) treatment was initiated, despite the high risk of failure (initiation 36 years after inhibitor onset, historical peak titer at 1800 BU, persistence of a plateau of 15–20 BU despite the absence of any stimulation with FVIII within the 3 last years). It started with an immunosuppressive drug, mycofenolate mofetil (Cellcept®) first given in may 2003 (no effect alone on inhibitor titer) and then in November 2003, infusions of a FVIII concentrate rich in von Willebrand factor, Factane® (LFB, Les Ulis, France) using 12,000 IU/day (150 IU/kg) of FVIII. The inhibitor peaked at 520 BU on D19 and was 0.5 BU by May 2004. Thus, the FVIII dosage was progressively reduced to 7000 IU/day. In July 2005, 24 hours after a 7000 IU FVIII infusion, inhibitor level was 0.7 BU, the residual FVIII level was 0.04IU/ml with a recovery of FVIIIc of 1.37%/IU/kg infused and a half-life of 4.9 hours. No significant change in the immunophenotype of peripheral blood lymphocytes was observed during this course. The patient’s quality of life was dramatically improved, with no hospitalization required and no bleeding episode observed within the 16 last months. The patient can now stand and has returned to his previous social activities. These preliminary results show that this IT treatment, performed despite a high theoretical risk of failure, resulted in this patient in a dramatic clinical improvement, even though biological criteria of success have not yet been achieved. The respective roles of the type of FVIII concentrate, and of immunosuppression remain to be assessed, as well as the cost/benefit analysis on a longer follow-up period.

Prophylactic Use of rFVIIa in a Young Hemophilia A Patient with Factor VIII Inhibitor.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 4068-4068
Author(s):  
Annie Borel-Derlon ◽  
Mounia Slaoui ◽  
Philippe Gautier ◽  
Patricia Guillon
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Immune Tolerance ◽  
Hemophilia A ◽  
Factor Viia ◽  
High Dose ◽  
Factor Viii Inhibitor ◽  
On Demand ◽  
Viii Inhibitor

Abstract The prevention of bleeding by prophylaxis regimen particularly for joint rehabilitation, could be considered a more effective treatment for hemophilia patients. In hemophiliacs with factor VIII inhibitor (F VIII inh) prophylaxis is not generally proposed because the bypassing agents for these patients may be less effective than F VIII concentrates. We report the regimen and results of a 6 months rFVIIa (Recombinant factor VIIa) prophylaxis, in a young hemophilia A patient (4 years old), with F VIII inh and immune tolerance induction (ITI) treatment and compared, with rFVIIa, the on demand treatment results for the 6 months prior to prophylaxis. After 2 years of a high dose regimen ITI, the FVIII inh titer was less than 50 BU and the immune tolerance treatment remains on going. Due to the development of a right knee target joint the rFVIIa prophylaxis was decided as an active rehabilitation approach to prevent the development of chronic arthropathy as well as to improve the quality of life of the child. During the 6 months period, prior to the initiation of rFVIIa prophylaxis 22 bleeds occurred i.e., 9 right knee hemarthrosis and 13 other joint bleedings and hematoma including elbow, wrist, ankle, foot, arm and chest. These bleeds were all treated with rFVIIa with a dose ranging from 100 to 200 μg/kg depending on the severity of the episodes and the duration of treatment ranged from 1 to 8 days. After 6 recurrent right knee hemarthrosis, a lavage of the joint was performed and prophylaxis with rFVIIa was subsequently initiated. A 120 μg/kg rFVIIa injection was performed 3 times a week concomitantly with the ITI treatment infusion and just before the physiotherapy course. During the 6 months of prophylaxis regimen we observed 9 bleeds with 3 major post traumatic bleedings which were treated by one 200 μg/kg/day rFVIIa injection which was resolved in one to three days. This prophylaxis treatment was effective for the arthropathy evolution and permitted the patient to return to school on a regular basis compared to the previous year. The total dose of on demand rFVIIa treatment used before prophylaxis was 458 mg/6 months. This amount decreased by 25% during the six months of prophylaxis with rFVIIa to reach 343 mg. The results of this significant observation led us to conclude that rFVIIa could be effectively used as prophylactic treatment in patients with FVIII inh and administered safely via a portacath device even in cases of high doses, as demonstrated in this young patient. This prevention approach resulted in a decrease of bleeding episodes, injections, and a significant improvement in the quality of life.

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