Long-Term Prophylaxis with Activated Recombinant FVII in Children with Hemophilia a and Inhibitor, Receiving Treatment with ITI Protocol

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4980-4980 ◽  
Author(s):  
Ekaterina Shiller ◽  
Victor Petrov ◽  
Pavel Svirin ◽  
Vladimir Vdovin ◽  
Igor Koltunov ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Factor Viii ◽  
Hemophilia A ◽  
Treatment Time ◽  
Conflicts Of Interest ◽  
Factor Vii ◽  
Treatment Level ◽  
Factor Viii Inhibitor ◽  
Bleeding Episodes

Abstract Background: Recent studies have shown that addition of bypassing agents to immuno-tolerance induction (ITI) protocol for patients with hemophilia A and inhibitor results in better control of bleeding episodes and improves quality of life. Few publications have addressed prophylactic usage of recombinant factors VIIa in these settings. Due to relatively low infusion volume, convenience of administration and high efficacy rFVIIa - Coagil-VII seems to be especially reasonable for ITI protocol. Aim: To assess the efficacy and safety of rFVIIa - Coagil-VII (SJC "GENERIUM", Russia) for prophylactic use during ITI protocol in patients with hemophilia A and inhibitor. Methods: Seven patients aged between 2 to 7 years with severe hemophilia A and inhibitor have been treated with ITI protocol using plasma derived factor VIII with von Willebrand factor. Seven of them simultaneously received treatment with Coagil-VII in individual doses (100-250 mkg/kg) and regimens (every 12 - 48 hours). When inhibitor reached level of 3 BU (Bethesda Unit) either dose or frequency of Coagil-VII administration were gradually reduced. After 1 BU the patients were given factor VIII only. Number and severity of bleeding events were assessed. Results: Five patients with high responding inhibitors and poor prognosis (history of high titer of factor VIII inhibitor, prolonged time between first inhibitor appearance and the beginning of ITI) received Coagil-VII in high doses 150 - 250 mkg/kg every 12-24 hours in 1-4 years. At time of booster effect titer of inhibitors reached 92 -16 000 BU. One of patients had ITI failure because of interruption of protocol, while 4 patients continue treatment. Level of 3 BU was reached by 4 patients at 40, 12, 35, and 3 months of treatment. Level of 1 BU was reached by 2 patients at 6 and 42 months of treatment. Significant clinical effect was achieved after 6 months of treatment. Time of bleeding episode was decreased from 7 (±2) to 2 (±1) days. Total number of hemorrhagic events, including hemarthrosis, hematomas and bleedings decreased by 3,7 fold (3,7 events per patient-month during first 6 months versus 1,0 events per patient-month). Only 1 hospital admission with bone fracture was recorded. All children have an active lifestyle and attend school. Two patients with low responding inhibitor and good prognosis received Coagil-VII in low doses 90 - 170 mkg/kg every 24-48 hours. Maximal titer of inhibitor was 1.3 - 1.9 BU. Both patients completed treatment with Coagil-VII in 2 and 4 months and continue ITI protocol and both achieved undetectable level of inhibitor. No bleeding episodes were recorded in these patients since the beginning of treatment. There was no clinical or laboratory evidence of thrombosis, thrombocytopenia, or disseminated intravascular coagulation. Conclusion: We report our experience of prolonged (2 months - 4 years) prophylactic treatment with recombinant activated factor VII (rFVIIa) - Coagil-VII in patients with hemophilia A and inhibitor. This prophylaxis is efficacious when doses and treatment regimens are individually determined. This approach results in reduction of bleeding episodes in all patients, as well as increase of quality of life. No any adverse events (AE and SAE) with prolonged use of Coagil-VII have been registered so far. Disclosures No relevant conflicts of interest to declare.

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.

scholarly journals Postpartum Acquired Hemophilia: A Rare Cause of Postpartum Hemorrhage

2013 ◽  
Vol 2013 ◽  
pp. 1-2 ◽  
Author(s):  
Srikanth Seethala ◽  
Sumit Gaur ◽  
Elizabeth Enderton ◽  
Javier Corral
Keyword(s):  
Factor Viii ◽  
Hemophilia A ◽  
Factor Vii ◽  
Normal Vaginal Delivery ◽  
Factor Viii Inhibitor ◽  
Activity Levels ◽  
Factor Viii Activity ◽  
Acquired Hemophilia A ◽  
Acquired Hemophilia ◽  
Viii Inhibitor

A 36-year-old female started having postpartum vaginal bleeding after normal vaginal delivery. She underwent hysterectomy for persistent bleeding and was referred to our institution. An elevation of PTT and normal PT made us suspect postpartum acquired hemophilia (PAH), and it was confirmed by low factor VIII activity levels and an elevated factor VIII inhibitor. Hemostasis was achieved with recombinant factor VII concentrates and desmopressin, and factor eradication was achieved with cytoxan, methylprednisolone, and plasmapheresis.

Acquired Factor VIII Inhibitor: A Single Institution Experience With 62 Patients

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 3610-3610
Author(s):  
Ewa M. Wysokinska ◽  
Ramila Mehta ◽  
Diane Grill ◽  
Rajiv K. Pruthi
Keyword(s):  
Factor Viii ◽  
Hemophilia A ◽  
Conflicts Of Interest ◽  
Post Partum ◽  
Rare Condition ◽  
Factor Viii Inhibitor ◽  
Remission Rates ◽  
Autoimmune Conditions ◽  
Viii Inhibitor ◽  
Acquired Factor Viii Inhibitor

Abstract Background Acquired Factor VIII inhibitor or autoimmune hemophilia A (AHA), has an estimated incidence of up to 1.5 cases per million/year and may result in severe hemorrhagic complications and death. Approximately 50% of cases have an underlying condition such as malignancies, autoimmune disorders and post-partum state. AHA should be suspected in any patient presenting with unexplained bleeding and an inhibited aPTT. Management consists of maintaining hemostasis and elimination of the inhibitor, however management is not standardized. We present 62 cases of AHA managed at Mayo Clinic Rochester, over the course of 36 years. We also analyzed whether aPTT at presentation correlated with the strength of inhibition measured by Bethesda Titer. Methods After IRB approval, medical records of patients with AHA were reviewed and all clinical data collected. Cumulative incidence of death was estimated by Kaplan-Meier analysis. Spearman correlation was used to calculate relation of APTT to Bethesda titer. Results Between 1976 and 2012, we identified 62 patients (male: 35), with a median age at diagnosis of AHA 69 years (mean 64, range 20-86). Clinical presentation consisted of extensive ecchymoses (n=40, 64%) in majority of cases. 29/62 (47%) patients had at least 1 identifiable predisposing condition with 12/62 (19%) patients with an underlying malignancy and 16/62 (26%) with underlying autoimmune conditions. Median Bethesda titer was 29 (range 1 to 1178). Bethesda titer was not related to the number or duration of hospitalizations. Most (69%) patients had at least one hospitalization and 12 (19%) had more than one hospitalization for bleeding complication. Inpatient therapy for bleeding consisted most commonly of FEIBA in 21 pts (34%) and rFVIIa in 6 pts (10%). Prednisone was the most common immunosuppressant used in 54 (87%) patients while Rituximab was used in 11 (18%) patients. Of 32 patients with available follow up labs most (69%) achieved remission. There was no difference in remission rates between patients treated or not treated with Rituximab (p=0.1735). Conclusion Acquired Hemophilia A is a rare condition with very heterogenous presentation. It affects mostly older male patients who present with ecchymoses and elevated APTT. The degree of APTT prolongation at the time of diagnosis does not correlate with the strength of the Bethesda titer and should not guide choice of therapy in a patient presenting with an acute bleed. Rituximab use in the 11 patients treated at Mayo did not seem to influence remission rates or survival. Disclosures: No relevant conflicts of interest to declare.

Inhibitors in mild/moderate haemophilia A: An update

2006 ◽  
Vol 96 (08) ◽  
pp. 113-118 ◽  
Author(s):  
Gian Salvagno ◽  
Giuseppe Lippi ◽  
Massimo Franchini
Keyword(s):  
Factor Viii ◽  
Hemophilia A ◽  
Tolerance Induction ◽  
Factor Vii ◽  
Factor Viii Inhibitor ◽  
Increased Risk ◽  
Bypassing Agents ◽  
The Many ◽  
Activated Prothrombin Complex Concentrates ◽  
Optimal Approach

SummaryThe development of inhibitors in patients with mild/moderate hemophilia A is an increasingly recognized occurrence and is manifested by the patients’ bleeding pattern becoming more severe. Inherited (hemophilia genetic mutations) and acquired (type and delivery of factor VIII replacement therapy) factors have been associated with an increased likelihood of developing factor VIII inhibitors. Although the use of bypassing agents (i.e. activated prothrombin complex concentrates and recombinant factor VII activated) has been demonstrated to be effective in controlling bleeding episodes in patients who develop factor VIII inhibitors, the limited data available in the literature are insufficient to determine the optimal approach to the eradication of inhibitors (i.e. immune tolerance induction, immunosuppression or both) for this group. Particular attention should be directed to the prevention of this complication in those patients with mild/moderate hemophilia recognized to be at increased risk of developing a factor VIII inhibitor. In conclusion, large prospective trials are warranted in order to elucidate the many still unclear pathogenic and therapeutic aspects of the development of inhibitors in patients with mild/moderate hemophilia A.

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.

A Case of Acquired Hemophilia Post Treatment with Recombinant Human Activated Protein C (rh APC).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 4021-4021
Author(s):  
Yelena Patsiornik ◽  
Archana Maini
Keyword(s):  
Factor Viii ◽  
Factor Vii ◽  
Factor Viii Inhibitor ◽  
Factor Xii ◽  
Operating Table ◽  
Apache Score ◽  
Acquired Hemophilia ◽  
Drotrecogin Alfa Activated ◽  
Bleeding Episodes ◽  
Viii Inhibitor

Abstract A 87-year old male without a diagnosis of hemophilia presented to the ER with a large shoulder hematoma, developing after a minor fall. Activated partial thromboplastin time (aPTT) was 79.4 sec, not correcting on mixing studies. Factor assays showed a factor VIII of 11.9 %, factor IX of 74.7 %, factor XI of 82.2 % and factor XII of 65.5 %. Anticardiolipin IgG, IgM antibodies and lupus anticoagulant were negative. Factor VIII inhibitor was found and initially measured at 14 Bethesda Unit (BU). A diagnosis of Acquired Hemophilia was made and the patient was treated with recombinant factor VII (rVIIa) and factor VII concentrates, FFP, PRBC transfusions and steroids. The patient had 4 bleeding episodes during hospitalization. First episode was not treated because of the lack of correct diagnosis. However, rVIIa was administered for all bleeding episodes and prior to surgical procedures like tracheostomy. Interestingly, despite immunosupression with steroids, the inhibitor titer did not decrease, rather it displayed a greater variation in the BU values ranging from 14 to 67. The patient bled unpredictably at different titers of inhibitor, without any concordance between the bleeding or the aPTT or the BU value. Finally,he succumbed to uncontrolled bleeding from the tracheostomy site and expired on operating table on day 30 of his fateful admission. This is a unique case of Acquired Hemophilia as 8 months ago this patient had received rhAPC, also known as Drotrecogin Alfa Activated (DAA) for severe sepsis with APACHE score of 25. DAA has a newly established therapeutic role in the inhibitor of factors Va and VIIIa, limiting the thrombotic effect and thus the mortality of sepsis. However, cost-effectiveness and safety of DAA remain somewhat controversial. Despite its recent entry into the medical armamentarium of our fight against sepsis, there exist several references in the published literature about the need of a confirmatory prospective trial about its role even in patients with a high APACHE score. Although neutralizing antibody against APC have not been detected in any patient and there are no published reports about DAA induced antibodies to factor VIII, it remains a viable hypothesis nonetheless. Unknown mechanisms such as exposure of new epitopes of factor VIII degraded by rhAPC could precipitate factor VIII antibodies. Further, the unpredictable titer of factor VIII inhibitor may be secondary to the unique mode of antibody formation in this instance. Lack of Correlation between Factor VIII inhibitor Titers and Clinical Sequelae Dates PTT PT Hb/Ht F VIII inhibitor(BU) Bleeding Episodes 2/01/05 151.4 32 7.1/22.4 On admission: soft tissue hematoma 2/02/05 79.4 14.8 6.2/18.2 14 2/04/05 84.6 16.2 9.3/27.7 29.8 2/05/05 75.2 10 11/32.9 2/06/05 74.8 16.1 9.8/29.7 2/07/05 90.4 17.5 9.2/26.5 67 2/08/05 162.0 17.0 8.8/26.7 GI bleeding, hematuria 2/10/05 97.4 14.1 7.5/22 2/13/05 119 14.6 7.4/22.7 3/01/05 113.8 14.1 9.4/29.8 35 3/02/05 9.2/28.8 Hematuria, rectal bleeding 3/03/05 86.2 12 7/21.2 Bleeding from tracheostomy site

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 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

Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group

Blood ◽  
2003 ◽  
Vol 102 (7) ◽  
pp. 2358-2363 ◽  
Author(s):  
Alessandro Gringeri ◽  
Lorenzo G. Mantovani ◽  
Luciana Scalone ◽  
Pier Mannuccio Mannucci ◽  
Keyword(s):  
Quality Of Life ◽  
Rare Disease ◽  
Rare Complication ◽  
Cost Of Care ◽  
Factor Vii ◽  
Recombinant Activated Factor Vii ◽  
Activated Factor Vii ◽  
Patients With Diabetes ◽  
Bleeding Episodes

Abstract Inhibitors in patients with hemophilia are a rare complication of a rare disease causing pain and disability in patients and impairment to the quality of their lives. Recent advances in treatment have brought improvements, but they have done so by absorbing larger amounts of financial resources. This study involved 52 Italian patients with hemophilia with high-responding inhibitors who were longitudinally observed for 18 months to evaluate concomitantly cost of care and quality of life. Overall, 0.6 bleeding episodes per patient per month were recorded. This frequency of events was lower than that reported in other cohorts of patients with hemophilia who were not taking inhibitors. The average monthly cost of care was, in euros, €18 000 (US $18 000) per patient, mainly because of treatment products. Recombinant activated factor VII, mostly used for orthopedic surgery, represented 50% of the expenses. Quality of life, measured through validated questionnaires, was similar to that of patients with severe hemophilia without inhibitors. In particular, physical quality of life was similar to that in patients with diabetes and on dialysis, whereas mental quality of life was comparable to that in the general population. This study shows that hemophilia complicated by inhibitors, a prototype of rare disease, requires high amounts of resources for management that provides a satisfactory quality of life. (Blood. 2003;102:2358-2363)

scholarly journals The Influence of Personalized 2-Year Physiotherapy on Musculoskeletal Health and Quality of Life in Severe Hemophilia Patients with Inhibitor - Interim Analysis after 1 Year of the Program

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 36-36
Author(s):  
Maria Helena Podolak-Dawidziak ◽  
Janusz Zawilski ◽  
Mariola Bober ◽  
Ewa Stefanska-Windyga ◽  
Anna Buczma ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Replacement Therapy ◽  
Hemophilia A ◽  
Conflicts Of Interest ◽  
Exercise Program ◽  
Rehabilitation Program ◽  
Severe Hemophilia ◽  
Musculoskeletal Health ◽  
Positive Effects

Development of factor VIII (FVIII) inhibitor is a serious complication of factor replacement therapy observed in about 15-30% of severe hemophilia A (HA) patients. Inhibitors interfere with the treatment by neutralizing intravenously injected FVIII. Although bleeding rate is not increased in severe HA patients who developed the inhibitor, the inhibitors reduce the efficacy of factor replacement therapy during bleeding episodes and in consequence accelerate the progression of hemophilic arthropathy. In Poland, prophylaxis in hemophilia with inhibitors has been introduced in 2016, and therefore many HA patients with inhibitor still suffer from particularly severe arthropathy, and have a reduced quality of life. The aim of the study was to evaluate the musculoskeletal health and quality of life in patients with severe hemophilia A with inhibitor before initiation of personalized physiotherapy and after 2 years of the personalized physiotherapeutic program. The study was performed in 24 persons with severe HA with inhibitor (mean age 42.1 years, median age 42 years) receiving prophylaxis regimen with aPCC 85 ± 15 IU/kg 3 times a week. At the beginning of the project, the patients were examined by hematologist and physiotherapist. Joint condition was evaluated using the Hemophilia Joint Health Score (HJHS) and magnetic resonance imaging of the most affected joint. Quality of life was evaluated using EQ-5D scale (EuroQoL). aPCC dosing schedule in context of rehabilitation program was adjusted by the hematologist. Each patient was enrolled to a personalized exercise program developed based on age, general condition, stage of arthropathy, date of the last bleeding episode, and a history of physiotherapy. The program consisted of weekly 60 to 90 minutes rehabilitation sessions at home, and participated in four 5-day rehabilitation camps with 5 hours of rehabilitation sessions a day organized in Poznan after 6, 12, 18 and 24 months of the program. All patients were examined by hematologist, and the rehabilitation was provided only to the patients on regular prophylaxis with aPCC provided that a dose of aPCC was always administered 1 hour before the initiation of rehabilitation session. HJHS varied among the participants, with mean value at baseline 34.2 (median 36.5). Baseline mean EQ-5D score was 54.2 (median 60). Evaluation at month 12 was performed in 18 participants (2 patients withdrawn from the program, and 4 patients could not participate in the evaluation due to COVID-19 pandemics). The results obtained at month 12 show a significant improving the quality of life and musculoskeletal health of patients with HA after combined prophylaxis and personalized rehabilitation. Mean HJHS decreased from 34.2 to 30.4 points (median from 36.5 to 31 points). An average 3.8 point decrease confirms positive effects of long-term aPCC prophylaxis with personalized, regular rehabilitation. Increase in mean EQ-5D score from 54.2 to 70.1 (median from 60 to 72) show enhancing patients' mood resulted from the improved health status and regained capability to conduct more normal lifestyle. After the first year of the project we can conclude that regular prophylaxis with aPCC and personalized, once-a-week rehabilitation program positively influence joint status and quality of life of adult HA patients with inhibitor complicated with significant arthropathy. Regular rehabilitation may slow down the progression of arthropathy due to the choice of the exercise program appropriate for each patient. These positive effects justify the continuation of the project in patients with hemophilia A with inhibitor. Figure Disclosures No relevant conflicts of interest to declare.

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