A pediatric patient with sickle cell disease may be a candidate for a bone marrow transplant. Walters et al developed guidelines to assist in patient selection. The authors are from multiple hospitals in North America and Europe..
Requirements:
(1) age < 16 years
(2) one of the following (sickling hemoglobinopathy)
(2a) SS disease (sickle cell anemia)
(2b) SC disease (sickle cell hemoglobin C)
(2c) sickle beta-thalassemia
(3) an HLA identical related donor
(4) performance status >= 70% (by Karnofsky or Lansky criteria, see Chapter 1)
(4) one or more significant complications
(5) no contraindications
|
Parameter |
Indication |
Contraindication |
|
chronic sickle lung disease |
Stage 1 or 2 |
Stage 3 or 4 |
|
acute chest syndrome
|
recurrent hospitalization or exchange transfusion |
|
|
CNS |
stroke lasting > 24 hours |
severe impairment (other than hemiplegia alone) |
|
neuropsychiatric status |
impaired, with abnormal MRI scan |
|
|
veno-occlusive disease (VOD) |
recurrent for several years |
|
|
renal impairment |
moderate |
severe (GFR < 30% of predicted) |
|
proteinuria |
moderate to severe |
|
|
proliferative retinopathy |
bilateral, with major visual impairment affecting one or both eyes |
|
|
bone disease |
osteonecrosis affecting multiple joints |
|
|
antibodies from RBC alloimmunization |
>= 2 |
|
|
HIV disease |
|
positive |
|
compliance with medical instructions |
|
poor |
|
liver disease |
|
acute hepatitis, chronic fibrosis or cirrhosis |
|
priapism (in males) |
recurrent |
|
where:
• Some contraindications are potentially reversible (acute hepatitis, etc).
Specialty: Clinical Laboratory, Hematology Oncology, Surgery, general, Genetics
