Guidelines for the management of
of APCC for management of joint bleeding
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of APCC for management of joint bleeding has been shown to be essentially equivalent (Level 2) [69]. 9. Notably, however, some patients respond better to one agent than the other, highlighting the need to individualize therapy. (Level 2) [69,70] 10. An anamnestic immune response should be expected in patients with hemophilia B and a FIX inhibitor treated with prothrombin complex concentrates – whether activated or not – since these concentrates all contain FIX. 11. On the other hand, the risk of anamnesis in patients with hemophilia A and an inhib- itor treated with a(n) (activated) prothrombin
COMPLICATIONS OF HEMOPHILIA 61 complex concentrate will vary depending on the concentrate and its content of FVIII, which is generally minimal. It is estimated that APCC leads to an anamnestic response in approximately 30% of FVIII inhibitor patients. 12. Although there has been interest in the use of immunosuppressive therapies in patients with inhibitors, their role is not yet defined, and there is no consensus as to whether they have a place in the management of these patients.
1. Up to 50% of hemophilia B patients with inhibi- tors may have severe allergic reactions, including anaphylaxis, to FIX administration. Such reac- tions can be the first symptom of inhibitor development. 2. Newly diagnosed hemophilia B patients, partic-
1. In patients with severe hemophilia A, eradica- tion of inhibitors is often possible by immune tolerance induction (ITI) therapy. (Level 2) [73,74] 2. Before ITI therapy, high-responding patients should avoid FVIII products to allow inhibitor titres to fall and to avoid persistent anamnestic rise. As noted, some patients may develop an anamnestic response to the inactive FVIII mole- cules in APCC as well. (Level 2) [75] 3. Optimal regimen (product or dose) for ITI remains to be defined. An international trial comparing 50 IU/kg three times a week to 200 IU/kg daily was recently stopped due to safety concerns (higher number of intercurrent bleeds) in the low-dose arm pending detailed analysis and interpretation of the data [76]. 4. Response to ITI may be less favourable in patients with moderate/mild hemophilia [63]. 5. Experience with ITI for hemophilia B inhibitor patients is limited. The principles of treatment in these patients are similar, but the success rate is much lower, especially in persons whose inhib- itor is associated with an allergic diathesis. 6. Hemophilia B inhibitor patients with a history of severe allergic reactions to FIX may develop nephrotic syndrome during ITI, which is not always reversible upon cessation of ITI therapy. Alternative treatment schedules, including immunosuppressive therapies, are reported to be successful [77]. Patients switching to new concentrates 1. For the vast majority of patients, switching prod- ucts does not lead to inhibitor development. 2. However in rare instances, inhibitors in previ- ously treated patients have occurred with the introduction of new FVIII concentrates. 3. In those patients, the inhibitor usually disappears after withdrawal of the new product. 4. Patients switching to a new factor concentrate
6.3 Transfusion-transmitted and other infection-related complications 1. The emergence and transmission of HIV, HBV and HCV through clotting factor products resulted in high mortality of people with hemo- philia in the 1980s and early 1990s [78,79]. 2. Many studies conducted all over the world indi- cate that HIV, HBV, and HCV transmission through factor concentrate has been almost completely eliminated [80,81]. GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 62 3. This is a result of the implementation of several risk-mitigating steps, which include careful selec- tion of donors and screening of plasma, effective virucidal steps in the manufacturing process, and advances in sensitive diagnostic technologies for detection of various pathogens [82]. 4. Recombinant factor concentrates have been adopted over the past two decades, particularly in developed countries. Recombinant products have contributed significantly to infection risk reduction. 5. The new challenge remains emerging and re-emerging infections, many of which are not amenable to current risk reduction measures. These include the non-lipid enveloped viruses and prions, for which diagnosis and elimination methods are still a challenge [81,83,84]. 6. As new treatments are continually emerging in this rapidly changing field, transfusion-trans- mitted infections in people with hemophilia are best managed by a specialist. Principles of management of HIV infection in hemophilia 1. Knowledge and expertise in the treatment of HIV-infected people with hemophilia is currently limited to case series and reports. HIV treatment in people with hemophilia is therefore largely informed by guidelines used in the non-hemo- philia population. 2. As part of the hemovigilance program, all people with hemophilia treated with plasma- derived products that are not adequately virus-inactivated should be tested for HIV at least every 6-12 months and whenever clini- cally indicated. (Level 4) [85] 3. The diagnosis, counselling, initiation of treat- ment, and monitoring of HIV, as well as the treatment of HIV-associated complications in infected people with hemophilia, should be the same as in the non-hemophilic population. (Level 2) [86,87] 4. None of the currently available classes of anti- HIV drugs are contraindicated in people with hemophilia. (Level 5) [88-90] Principles of management of HCV infection in hemophilia 1. Assessment of HCV in people with hemophilia should include: ■ anti-HCV serology to determine exposure ■ HCV polymerase chain reaction (PCR) in those who are anti-HCV positive ■ HCV genotyping in those who are HCV PCR positive
■ liver function tests and non-invasive assess- ment of fibrosis and liver architecture 2. The current standard of treatment for HCV
3. New antiviral therapies, in combination with these drugs, may improve sustained virologic response rates [97]. 4. HCV genotype 1 and HIV coinfection predict poorer response to anti-HCV therapy. 5. Where HCV eradication cannot be achieved,
1. All people with hemophilia treated with plasma- derived products that are not adequately virus-inactivated should be screened for hepa- titis B antigen and anti-hepatitis B at least every 6-12 months and whenever clinically indicated. (Level 4) [99] 2. Active HBV infection should be managed as per local infectious disease guidelines and protocols. 3. Those without HBV immunity should be given the anti-HBV vaccine. Protective COMPLICATIONS OF HEMOPHILIA 63
vaccination. (Level 4) [99-101] 4. People with hemophilia who do not seroconvert should be revaccinated with double the hepa- titis B vaccine dose. (Level 4) [99,102] Principles of management of bacterial infection in hemophilia 1. The risk factors for bacterial infections in people with hemophilia are venous access catheter insertion, surgical arthroplasty, and other surgical interventions [103-105]. 2. In general, joint aspiration to treat hemarthrosis should be avoided, unless done early under appro- priate cover of factor replacement and with strict aseptic precautions to prevent infection [106,107]. 3. Bleeding is likely to delay healing and worsen infection and should therefore be well controlled [108].
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intensity o f factor r eplacement 5 0
Treatment of pain and serious bleeding Short-term prophylaxis Improvement of target joints Tertiary prophylaxis
(after onset of joint disease) Improves normal activities of daily life Secondary prophylaxis
(after second joint bleed) Minimal musculoskeletal disease Primary prophylaxis (before second joint bleed) Near normal musculoskeletal & psycho-social development Adapted from Blood Transfus 2008 Sep;6 Suppl 2:s4-11
GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA 70 4. Table 7-1 and Table 7-2 present commonly followed guidelines on plasma factor peak levels and duration of replacement that reflect the different practices in countries where there is no significant resource constraint (Table 7-1) and countries where treatment products are limited (Table 7-2). 5. With the lower doses for treating musculoskel- etal bleeds listed in Table 7-2, it may only be possible to avoid major target joints and crip- pling deformities. 6. Higher doses listed in Table 7-1 have been shown to avoid joint damage, but the optimal dose needed to achieve this remains to be defined. 7. Observational studies documenting the musculo- skeletal outcome of doses and protocols of factor replacement are extremely important in defining these issues. 8. Doses for prophylactic replacement of factor concentrates vary between different countries and also among centres in the same country. 9. Commonly-used dosage for prophylactic factor
10. In situations where there are greater constraints on supply of factor concentrates, prophylaxis may be initiated with lower doses of 10-20 IU/kg 2-3 times per week. (Level 2) [4,5] PLASMA FACTOR LEVEL AND DURATION OF ADMINISTRATION 71 TABLE 7-1: SUGGESTED PLASMA FACTOR PEAK LEVEL AND DURATION OF ADMINISTRATION (WHEN THERE IS NO SIGNIFICANT RESOURCE CONSTRAINT) [6] TYPE OF HEMORRHAGE HEMOPHILIA A HEMOPHILIA B DESIRED LEVEL
(IU/DL) DURATION (DAYS) DESIRED LEVEL
(IU/DL) DURATION (DAYS) Joint 40–60
1–2, may be longer if response is inadequate 40–60 1–2, may be longer if response is inadequate Superficial muscle/no NV compromise (except iliopsoas) 40–60
2–3, sometimes longer if response is inadequate 40–60 2–3, sometimes longer if response is inadequate Iliopsoas and deep muscle with NV injury, or substantial blood loss
■ initial 80–100 1–2
60–80 1–2
■ maintenance 30–60 3–5, sometimes longer as secondary prophylaxis during physiotherapy 30–60 3–5, sometimes longer as secondary prophylaxis during physiotherapy CNS/head
■ initial 80–100 1–7
60–80 1–7
■ maintenance 50 8–21
30 8–21
Throat and neck
■ initial 80–100 1–7
60–80 1–7
■ maintenance 50 8–14
30 8–14
Gastrointestinal
■ initial 80–100 7–14
60–80 7–14
■ maintenance 50 30
50 3–5
40 3–5
Deep laceration 50 5–7 40 5–7
Surgery (major)
■ Pre-op 80–100 60–80
■ Post-op 60–80 40–60
30–50 1–3
4–6 7–14
40–60 30–50
20–40 1–3
4–6 7–14
Surgery (minor)
■ Pre-op 50–80 50–80
■ Post-op 30–80 1-5, depending on type of procedure 30–80
1–5, depending on type of procedure NV; neurovascular
72 GUIDELINES FOR THE MANAGEMENT OF HEMOPHILIA TABLE 7-2: PLASMA FACTOR PEAK LEVEL AND DURATION OF ADMINISTRATION (WHEN THERE IS SIGNIFICANT RESOURCE CONSTRAINT) TYPE OF HEMORRHAGE HEMOPHILIA A HEMOPHILIA B DESIRED
LEVEL (IU/DL)
DURATION (DAYS) DESIRED
LEVEL (IU/DL)
DURATION (DAYS) Joint
10–20 1–2 may be longer if response is inadequate 10–20
1–2, may be longer if response is inadequate Superficial muscle/no NV compromise (except iliopsoas) 10–20 2–3, sometimes longer if response is inadequate 10–20
2–3, sometimes longer if response is inadequate Iliopsoas and deep muscle with NV injury, or substantial blood loss
■ initial 20–40 15–30
■ maintenance 10–20 3–5, sometimes longer as secondary prophylaxis during physiotherapy 10–20 3–5, sometimes longer as secondary prophylaxis during physiotherapy CNS/head
■ initial 50–80 1–3
50–80 1–3
■ maintenance 30–50 20–40
4–7 8–14
30–50 20–40
4–7 8–14
Throat and neck
■ initial 30–50 1–3
30–50 1–3
■ maintenance 10–20 4–7
10–20 4–7
Gastrointestinal
■ initial 30–50 1–3
30–50 1–3
■ maintenance 10–20 4–7
10–20 4–7
Renal 20–40
3–5 15–30
3–5 Deep laceration 20–40 5–7
15–30 5–7
Surgery (major)
■ Pre-op 60–80 50–70
■ Post-op 30–40 20–30
10–20 1–3
4–6 7–14
30–40 20–30
10–20 1–3
4–6 7–14
Surgery (minor)
■ Pre-op 40–80 40–80
■ Post-op 20–50 1–5, depending on type of procedure 20–50
1–5, depending on type of procedure NV; neurovascular
73 PLASMA FACTOR LEVEL AND DURATION OF ADMINISTRATION References 1. Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be in dividualized. Br J Haematol 1999 Jun;105(4):1109-13. 2. Blanchette VS. Prophylaxis in the haemophilia population. Haemophilia 2010;16(Suppl 5):181-8. 3. Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM; ESPRIT Study Group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost 2011 Apr;9(4):700-10. 4. Fischer K, van der Bom JG, Mauser-Bunschoten EP, Roosendaal G, Prejs R, Grobbee DE, van den Berg HM. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001 Sep;7(5):446-52. 5. Wu R, Luke KH, Poon MC, Wu X, Zhang N, Zhao L, Su Y, Zhang J. Low dose secondary prophylaxis reduces joint bleeding in severe and moderate haemophilic children: a pilot study in China. Haemophilia 2011 Jan;17(1):70-4. 6. Rickard KA. Guidelines for therapy and optimal dosages of coagulation factors for treatment of bleeding and surgery in haemophilia. Haemophilia 1995;1(S1):8–13. APPENDIX I: Oxfor
d Centr
e for Evidence-Based Medicine, 2011 Levels of Evidence
QUESTION STEP 1 (LEVEL 1*) STEP 2 (LEVEL 2*) STEP 3 (LEVEL 3*) STEP 4 (LEVEL 4*) STEP 5 (LEVEL 5) How common is the pr oblem? Local and curr ent random sample surveys (or censuses) Systematic r eview of surveys that allow matching to local cir cumstances** Local non-random sample** Case-series** n/a Is this
diagnostic
or monitoring test accurate?
(Diagnosis) Systematic r eview of cr oss sectional studies with consistently applied refer
ence standar d and
blinding Individual cr oss sectional studies with consistently applied r efer
ence standar d and blinding Non-consecutive studies, or studies without consistently applied r efer
ence standar
ds** Case-contr ol studies, or “poor or non-independent refer ence standar d** Mechanism-based r easoning What will happen
if we do not add a therapy? (Pr
ognosis) Systematic r eview of inception cohort studies Inception cohort studies Cohort study or contr ol arm of randomized trial* Case-series or case contr ol
studies, or poor quality pr ognostic cohort study** n/a Does
this
intervention help? (T reatment Benefits) Systematic r eview of randomized trials or
trials
Randomized trial or observational study with dramatic ef fect
Non-randomized contr olled
cohort/ follow-up study** Case-series, case-contr ol studies, or historically contr olled studies** Mechanism-based r easoning
What ar e the
COMMON har ms? (T
Systematic r eview of randomized trials, systematic review of nested case- contr ol studies, n -of-1 trial with the patient you ar e raising the question about, or observational study with dramatic ef fect Individual randomized trial or (exceptionally) observational study with dramatic ef fect
Non-randomized contr olled
cohort/ follow-up study (post-marketing surveillance) pr ovided ther e ar e suf
ficient numbers to rule out a common harm. (For long- term harms the duration of follow-up must be suf
ficient.)** Case-series, case-contr ol, or historically contr olled studies** Mechanism-based r easoning
What ar e the
RARE har
ms? (T reatment Harms) Systematic r eview of randomized trials or
trial
Randomized trial or (exceptionally) observational study with dramatic ef fect
Is this (early detection) test worthwhile? (Scr
eening) Systematic r eview of randomized trials Randomized trial Non -randomized contr olled cohort/follow-up study** Case-series, case-contr ol, or
historically contr olled
Mechanism-based r easoning
* Level may be graded down on the basis of study quality , impr
ecision, indir ectness (study PICO does not match questions PICO), because of inconsistency between studies, or because the absolute ef fect size is very small; Level may be graded up if ther e is a lar ge or very lar ge ef fect size. ** As always, a systematic r eview is generally better than an individual study . OCEBM Levels of Evidence W orking Gr oup*. “The Oxfor d 2011 Levels of Evidence”. Oxfor d Centr
e for Evidence-Based Medicine. http://www .cebm.net/index.aspx?o=5653 APPENDIX I: Oxfor
d Centr
e for Evidence-Based Medicine, 2011 Levels of Evidence
QUESTION STEP 1 (LEVEL 1*) STEP 2 (LEVEL 2*) STEP 3 (LEVEL 3*) STEP 4 (LEVEL 4*) STEP 5 (LEVEL 5) How common is the pr oblem? Local and curr ent random sample surveys (or censuses) Systematic r eview of surveys that allow matching to local cir cumstances** Local non-random sample** Case-series** n/a Is this
diagnostic
or monitoring test accurate?
(Diagnosis) Systematic r eview of cr oss sectional studies with consistently applied refer
ence standar d and
blinding Individual cr oss sectional studies with consistently applied r efer
ence standar d and blinding Non-consecutive studies, or studies without consistently applied r efer
ence standar
ds** Case-contr ol studies, or “poor or non-independent refer ence standar d** Mechanism-based r easoning What will happen
if we do not add a therapy? (Pr
ognosis) Systematic r eview of inception cohort studies Inception cohort studies Cohort study or contr ol arm of randomized trial* Case-series or case contr ol
studies, or poor quality pr ognostic cohort study** n/a Does
this
intervention help? (T reatment Benefits) Systematic r eview of randomized trials or
trials
Randomized trial or observational study with dramatic ef fect
Non-randomized contr olled
cohort/ follow-up study** Case-series, case-contr ol studies, or historically contr olled studies** Mechanism-based r easoning
What ar e the
COMMON har ms? (T
Systematic r eview of randomized trials, systematic review of nested case- contr ol studies, n -of-1 trial with the patient you ar e raising the question about, or observational study with dramatic ef fect Individual randomized trial or (exceptionally) observational study with dramatic ef fect
Non-randomized contr olled
cohort/ follow-up study (post-marketing surveillance) pr ovided ther e ar e suf
ficient numbers to rule out a common harm. (For long- term harms the duration of follow-up must be suf
ficient.)** Case-series, case-contr ol, or historically contr olled studies** Mechanism-based r easoning
What ar e the
RARE har
ms? (T reatment Harms) Systematic r eview of randomized trials or
trial
Randomized trial or (exceptionally) observational study with dramatic ef fect
Is this (early detection) test worthwhile? (Scr
eening) Systematic r eview of randomized trials Randomized trial Non -randomized contr olled cohort/follow-up study** Case-series, case-contr ol, or
historically contr olled
Mechanism-based r easoning
* Level may be graded down on the basis of study quality , impr
ecision, indir ectness (study PICO does not match questions PICO), because of inconsistency between studies, or because the absolute ef fect size is very small; Level may be graded up if ther e is a lar ge or very lar ge ef fect size. ** As always, a systematic r eview is generally better than an individual study . OCEBM Levels of Evidence W orking Gr oup*. “The Oxfor d 2011 Levels of Evidence”. Oxfor d Centr
e for Evidence-Based Medicine. http://www .cebm.net/index.aspx?o=5653 Document Outline
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