The purpose of this clinical trial is to evaluate the clinical and cost effectiveness of Haploidentical Stem Cell Transplantation (SCT) for adults with severe sickle cell disease (SCD), who have failed other therapies or are intolerant of existing therapies or require chronic transfusions to prevent on-going complications of SCD.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
120
Stem cell transplant from bone marrow or peripheral blood from haploidentical donor using standard nationally approved transplant procedure.
Standard medical care may include any currently available therapies for SCD patients. These may or may not include regular elective transfusion therapy or medications such as hydroxycarbamide.
King's College Hospital
London, United Kingdom
RECRUITINGTreatment failure or mortality
Treatment failure is defined as occurrence of vaso-occlusive crisis, or transfusion from 6 months post-randomisation.
Time frame: 24 months post-randomisation
Health related quality of life
Quality of life as measured by EQ-5D-5L
Time frame: At 3, 6, 9, 12, 15, 18, 21 and 24 months post-randomisation
All cause mortality
Death by any cause
Time frame: 24 months post-randomisation
Sickle Cell Disease-related mortality (excluding transplant related complications)
Death due to any sickle cell disease related cause
Time frame: 24 months post-randomisation
Sickle type haemoglobin percentage (HbS%)
Sickle type haemoglobin as measured by haemoglobin electrophoresis
Time frame: At 6, 12 and 24 months post-randomisation
Sickle cell disease related complications
Defined as transfusion requirement, painful VOC, stroke, pulmonary hypertension
Time frame: 24 months post-randomisation
Haemoglobin levels, Reticulocyte count, LDH, Bilirubin
Time frame: At 6, 12 and 24 months post-randomisation
Pulmonary Function
As measured by FEV1 %, FEV1/FVC ratio, TLCO %
Time frame: At 12 months and 24 months post-randomisation
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Renal Function
As measured by urea, creatinine and eGFR
Time frame: At 6, 12 and 24 months post-randomisation
Iron overload
As measured by Ferritin and FerriScan (R2-MRI)
Time frame: 24 months post-randomisation
Cardiac function and pulmonary hypertension
As measured by echocardiogram/TRV
Time frame: At 12 and 24 months post-randomisation
Cerebrovascular progression
As measured by clinical stroke or evidence of progression on MRI/MRA
Time frame: 24 months post-randomisation
Evidence of hepatic progression
As measured by liver function (ALT, AST, ALP, GGT, Bilirubin) and FibroScan
Time frame: 24 months post-randomisation
Percentage of participants requiring opioid use for pain related to vaso-occlusive sickle related crisis
Time frame: At 12 and 24 months post-randomisation