dose titration in a paediatric patient with sickle cell disease n.
Download
Skip this Video
Loading SlideShow in 5 Seconds..
Dose Titration in a Paediatric Patient with Sickle Cell Disease PowerPoint Presentation
Download Presentation
Dose Titration in a Paediatric Patient with Sickle Cell Disease

Loading in 2 Seconds...

play fullscreen
1 / 11

Dose Titration in a Paediatric Patient with Sickle Cell Disease - PowerPoint PPT Presentation


  • 145 Views
  • Uploaded on

Dose Titration in a Paediatric Patient with Sickle Cell Disease. Background. There is a significant correlation between the number of transfusions a patient receives and the degree of iron overload in sickle cell disease (SCD) that is reflected in organ failure 1

loader
I am the owner, or an agent authorized to act on behalf of the owner, of the copyrighted work described.
capcha
Download Presentation

PowerPoint Slideshow about 'Dose Titration in a Paediatric Patient with Sickle Cell Disease' - butch


An Image/Link below is provided (as is) to download presentation

Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author.While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server.


- - - - - - - - - - - - - - - - - - - - - - - - - - E N D - - - - - - - - - - - - - - - - - - - - - - - - - -
Presentation Transcript
background
Background
  • There is a significant correlation between the number of transfusions a patient receives and the degree of iron overload in sickle cell disease (SCD) that is reflected in organ failure1
  • To prevent neurologic complications, 10% of children with SCD begin monthly transfusions at a young age2,3

1. Ballas SK. Semin Hematol. 2001;38:30-36. 2. Adams RJ, et al. N Engl J Med.1998;339:5-11.

3. Adams RJ, et al. N Engl J Med. 2005; 353: 2769-2778.

background1
Background
  • Mild, nonprogressive increases in serum creatinine >33% from baseline (at 2 consecutive visits) have been observed in 36% of patients receiving deferasirox in clinical trials
  • In most cases (71%), these increases resolved spontaneously; the remainder (29%) required dose adjustment

Vichinsky E, et al. Br J Haematol. 2007;136:501-508.

patient presentation
Patient Presentation
  • 9-year-old African-American female patient diagnosed with sickle cell disease at birth
  • Patient experienced a stroke at 3 years of age and has been receiving transfusions of 1 unit of packed red blood cells per month since this event
  • Patient has never received chelation therapy
  • Patient presented with baseline liver iron concentration, assessed using a superconducting quantum interference device (SQUID), of 10.8 mg Fe/g dry weight and serum ferritin of 3210 ng/mL
question
Question

Should chelation therapy be initiated atthis point?

A. No, not until she is older

B. Yes, with desferrioxamine

C. Yes, with deferiprone

D. Yes, with deferasirox

treatment
Treatment
  • Chelation therapy should be initiated
    • Serum ferritin is above the recommended level of 1,000 ng/mL for initiation of treatment
  • Deferiprone is not indicated for iron overload in patients with sickle cell disease (SCD)
  • The need for subcutaneous administration also makes it less suitable for paediatric use than orally administered deferasirox, especially in this patient, who has a fear of needles
  • Deferasirox is approved for children ≥2 years of age and for patients with SCD
question1
Question

Deferasirox was initiated at 20 mg/kg/d. After 3 months of treatment, serum ferritin levels remained unchanged at around 3200 ng/mL. What is the next step?

A. Continue deferasirox at current dosage

B. Increase dosage of deferasirox

C. Consider switching to deferiprone

response to dose increase
Response to Dose Increase
  • Deferasirox dose was increased to 30 mg/kg/d
  • 9 months later
    • Liver iron concentration had decreased from 10.8 to 8.2 mg Fe/g dry weight
    • Serum ferritin levels had decreased from 3210 to 2560 ng/mL
increase in serum creatinine
Increase in Serum Creatinine
  • With increase of deferasirox to 30 mg/kg/d, serum creatinine also increased, exceeding baseline by >33% at 2 consecutive visits
    • These increases were above the upper limit of normal appropriate for a 9-year-old
  • Deferasirox dose was therefore reduced to 20 mg/kg/d at month 10
current status
Current Status
  • Patient remains on deferasirox 20 mg/kg/d and serum ferritin levels continue to fall
  • She has not experienced any further adverse events
  • Her growth and development are within normal limits
conclusions
Conclusions
  • In some paediatric patients, 20 mg/kg/d may be sufficient to decrease serum ferritin levels
  • Deferasirox dose should be reviewed regularly at 3- to 6-month intervals and adjusted according to trends in serum ferritin levels
  • Increases in serum creatinine >33% above baselinea can be managed by dose reduction of 10 mg/kg/d

a And above age-appropriate limit of normal in paediatric patients.