Mohammadreza Bordbar MD Pediatric Hematologist Associate professor of SUMS Qeshm island 22 feb 2018 Introduction Care for persons with SCD often lacks continuity Two SCDspecific diseasemodifying treatments HU blood transfusion still underutilized ID: 775091
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Slide1
Monitoring of treatment in sickle cell anemia
Mohammadreza
Bordbar
, MD
Pediatric Hematologist
Associate professor of SUMS
Qeshm
island, 22
feb
2018
Slide2Introduction
Care for persons with SCD often lacks continuityTwo SCD-specific disease-modifying treatments (HU, blood transfusion) still underutilizedUp-to-date clinical guidelines is a necessityNIH-sponsored SCD guidelines, “Evidence-Based Management of Sickle Cell Disease, Expert Panel Report 2014”
JAMA. 2014;312(10):1033-1048Am Fam Physician. 2015;92(12):1069-1076
Slide3Health maintenance
Prevention of invasive pneumococcal infection - oral penicillin prophylaxis at least up to age of 5 years - beyond the age of 5 if splenectomy or invasive pneumococcal infection - complete pneumococcal vaccination before discontinuation - at least 1 dose of PCV-13 in children 6-18 years with functional or anatomic asplenia
Slide4Screening for hepatitis C
Screen in patients at high risk for infection (multiple transfusion)
Slide5Screening for retinopathy
Dilated eye examination since the age of 10 yearsRescreen every 1-2 years if NL exam
Slide6Screening for risk of stroke
Screen with TCD annually starting at age 2 till the age of 16 yearsConsider regular transfusion in those with conditional (170-199cm/s) or elevated (≥200cm/s) TCDIn asymptomatic children with SCD, no need for MRI or CTIn asymptomatic adults with SCD, no need for TCD or MRI/CTDo not screen children with other SCD (S/β+ -thalassemia, HbSC)
Slide7Cardiovascular/ pulmonary screening
No need for ECG screening in asymptomatic children or adults
No need for PFT in asymptomatic children or adults
Doppler echocardiography, NT-pro-BNP, Right heart catheterization (RHC) in symptomatic patients for screening pulmonary hypertension
Slide8American thoracic society clinical practice guideline
Increased risk of mortality:Tricuspid regurgitant jet velocity (TRV) ≥ 2.5 m/secondNT-pro-BNP ≥ 160 pg/mlRHC-confirmed PH (resting mean PAP ≥ 25 mm Hg)
Am J Respir Crit Care Med. 2014 Mar 15; 189(6): 727–740.
Slide9Proposed algorithm for evaluation of pulmonary hypertension in SCD
Am J
Respir
Crit
Care Med. 2014 Mar 15; 189(6): 727–740
.
Slide10Managing chronic complications
Avascular necrosis:
- if intermittent or chronic hip pain, evaluate for AVN by
Hx
, PE, radiography
and MRI as needed
- treat with analgesics
- consult with physical therapy and orthopedic department
Slide11Renal complications
Regular assessment of creatinine, GFR
Screen annually for proteinuria with spot urine test since the age of 10 years
If proteinuria > 300mg/24
hr
, refer to a nephrologist for further evaluation
Initiate ACE inhibitor in adults with microalbuminuria or proteinuria without other apparent cause even with NL BP
Renal replacement therapy (dialysis, transplantation) if needed
Slide12Leg ulcers
Standard therapy (debridement, wet to dry dressing, topical agents)
Evaluate for osteomyelitis in chronic recalcitrant deep leg ulcers
Treat with local or systemic antibiotics if suspicious for infection and positive wound culture
Slide13Evidence-based recommendations for use of hydroxyurea therapy
JAMA. 2014;312(10):1033-1048
Slide14Adults with ≥3 moderate to severe pain crisis during a 12-mo period
Pains interfering with daily activities and QOLSevere or recurrent ACSSevere symptomatic chronic anemia interfering with daily activities or QOLAdults and children with chronic kidney disease taking EPO to improve anemiaInfants 9 mo or older, children and adolescents regardless of clinical severity to reduce complications (dactylitis, pain, ACS, anemia)Discontinue in pregnant or breastfeeding women
Slide15Monitoring HU therapy
Laboratory tests before starting therapy:
- CBC, diff;
Retic
count
- quantitative
Hb
F measurement
- renal and liver function tests
- pregnancy test for women
Slide16Initiating HU therapy
Baseline elevation of
Hb
F should not affect the decision to initiate HU
Counselling regarding contraception in both genders
o
f reproductive age
Starting dose for adults:
15mg/kg/d
round up to the nearest 500mg
5-10 mg/kg/d in adults with chronic kidney disease
Starting dose for infants & children:
20mg/kg/d
Slide17Monitoring hu therapy
CBC, diff &
R
etic
count every 4wks when adjusting dosage
Aim for a target
ANC≥2000/µl
Younger persons with lower baseline counts may safely tolerate ANC down to
1250/µl
Maintain
platelet ≥ 80,000/µl
Slide18If neutropenia or thrombocytopenia occurs
Stop HU temporarily
Monitor CBC, diff weekly
Restart HU at a dose 5mg/kg/d lower dose when
cytopenia
recovered
Slide19Dose escalation
Increase by
5mg/kg/d
every 8wk
Give until mild myelosuppression (ANC 2000-4000/µl) up to a maximum dose
35mg/kg/d
CBC, diff &
Retic
count every 2-3
mo
once stable dose established
Slide20Evidence-based recommendations for use of transfusion therapy
JAMA. 2014;312(10):1033-1048
Slide21Symptomatic severe ACS (O2 saturation <90% despite supplemental O2)
Symptomatic ACS and ↓Hb˃ 1g/dL from baseline (if baseline Hb˂ 9g/dL)Acute splenic sequestration plus severe anemiaAcute strokeHepatic sequestrationIntrahepatic cholestasisMultisystem organ failureAplastic crisisSymptomatic anemiaChild with TCD≥ 200cm/sAdults and children with previous clinically overt stroke
Slide22Simple vs exchange transfusion
Slide23Evidence-based recommendations against use of transfusion
Uncomplicated painful crisisPriapismAsymptomatic anemiaAcute kidney injury without multi-organ failureRecurrent splenic sequestration
JAMA. 2014;312(10):1033-1048
Slide24Precautions and monitoring
RBC phenotype matching to at least C, E, K antigensMonitor for delayed transfusion reactionsMonitoring serum ferritin quarterlyT2* MRI (the optimal frequency of assessment not established)Iron chelation if confirmed iron-overload (LIC ≥ 7mg Fe/g dry weight)
Med
Clin
N Am 101 (2017) 375–393
Slide25A summary of clinical trials in SCD (primary prevention of life-threatening infections)
Slide26primary & secondary prevention of stroke
Slide27hsct
Slide28Evidence-based strong recommendations with high-quality evidence (JAMA. 2014;312(10):1033-1048)
Slide29Thanks for your attention