Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies

Figures & data

Table 1 Treatment Options in Sickle Cell Disease

Therapy/Agent	Age at Which Approved for Use	Indications	Contra-Indication	Comments
Disease Modifying
Hydroxyurea	≥2 years (can start as early as 9 months)	Children, HBSS, HBSβ0	Pregnancy, breastfeeding	Requires frequent blood tests for monitoring of bone marrow depression, and needs dose reduction in patients with compromised renal function
Voxelotor	≥12 years	for increase of hemoglobin by 1% in any sickle cell disease	End stage renal disease Pregnancy and breast feeding	Only symptomatic treatment, no change in end organ damage Cost can be prohibitive for use
Crizanlizumab	≥16 years	Reduction of pain crises (if frequency ≥2 per year)	Pregnancy and breast feeding	Requires intravenous administration Only symptomatic treatment, no change in end organ damage Cost can be prohibitive for use
L-Glutamine	≥5 years	To reduce the severity and frequency of complications.	Pregnancy and breast feeding	No change in end-organ damage
Chronic Blood Transfusion	Any age	In children with abnormal transcranial doppler velocities, or history of stroke or evidence of cerebrovascular disease on magnetic resonance angiogram.	None	Concerns for iron overload, allo-immunization, hyperviscosity.
Curative
Hematopoietic Stem Cell Transplant	No specific age, but earlier age improves outcomes.	Stroke, recurrent VOC, Acute chest requiring exchange transfusion, osteonecrosis of multiple joints, sickle nephropathy, red cell alloimmunization (≥2 antibodies) on long term transfusion therapy	Nil	Availability of HLA-matched donor is a serious consideration, treatment related morbidities include including Graft versus host disease, infertility and risk of subsequent malignancy.

Notes: Modified with permission from Annals of Internal Medicine, Pecker LH, Lanzkron S, Sickle cell disease, 2021;174(1):Itc1–Itc16. Copyright © 2021 American College of Physicians. All Rights Reserved.2² Data from Krisnamurti L.^{340340340340340340340340340340340}

Figure 1 Review of literature and identification of studies.

Pecker LH, Lanzkron S. Sickle cell disease. Ann Intern Med. 2021;174(1):Itc1–Itc16. doi:10.7326/aitc202101190

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