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خرید پکیج
تعداد آیتم قابل مشاهده باقیمانده : 3 مورد

Disease-modifying therapies for sickle cell disease

Disease-modifying therapies for sickle cell disease
Therapy Hydroxyurea
(1998)
(Siklos, Hydrea)		 L-glutamine
(2017)
(Endari)		 Crizanlizumab
(2019)
(Adakveo)
Age All ages including infants 5 years and older* 16 years and older*
Route Oral (pill or liquid; liquid used for young children requires formulary preparation) Oral (powder added to food or beverages) Intravenous
Frequency Once daily Twice daily Once every 4 weeks (after initial dose week 0, 2, and 4)
Monitoring Frequent monitoring of CBC and dose titration, especially during drug initiation None None
Benefits
  • Improved life expectancy
  • Reduced acute pain episodes (4.5 versus 2.5)
  • Reduced vaso-occlusive complications including acute chest syndrome and stroke (selected individuals)
  • Reduced transfusion requirement
  • 25 years of safety and efficacy data
  • Reduced acute pain episodes (4 versus 3)
  • Can be combined with hydroxyurea
  • Good safety profile
  • Reduced in acute pain episodes (3 versus 1.6)
  • Can be combined with hydroxyurea
  • Good choice for severe and frequent pain episodes
Caveats
  • Hair loss
  • Gastrointestinal symptoms
  • Generally avoid prior to conception (males and females) and during first trimester of pregnancy
  • Use pharmacologic grade (not over-the-counter) L-glutamine
  • Mild gastrointestinal symptoms
  • Long-term efficacy data are lacking
  • Requires intravenous access and hospital infusions
  • Infusion reactions (rare)
  • Arthralgias and back pain
  • Nausea
  • May cause platelet clumping in samples collected in EDTA
These medications are intended to be started when the individual is well; they are not appropriate for treating acute pain. Individuals with sickle cell disease may consider the information in this table or other information along with their values and preferences when determining the best therapy to use. For each drug, the year the drug was approved by the US Food and Drug Administration (FDA) and associated brand names are listed in parentheses. Voxelotor was withdrawn from the market in 2024 due to an unfavorable risk-benefit ratio.

CBC: complete blood count; EDTA: ethylenediaminetetraacetic acid.

* Refers to age listed on United States product information. At times we use these drugs in selected younger patients (off-label).

¶ Expressed as annualized rates, compared with placebo.[1-4] None of the drugs have been directly compared with each other in a randomized trial.
References:
  1. Charache S, Terrin ML, Moore RD, et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med 1995; 332:1317.
  2. Niihara Y, Miller ST, Kanter J, et al. A Phase 3 Trial of L-glutamine in Sickle Cell Disease. N Engl J Med 2018; 379:226.
  3. Vichinsky E, Hoppe CC, Ataga KI, et al. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. N Engl J Med 2019; 381:509.
  4. Ataga KI, Kutlar A, Kanter J, et al. Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease. N Engl J Med 2017; 376:429.
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