Avascular Necrosis of Bone, Sickle Cell Disease
Conditions
Keywords
Cell therapy, Sickle cell disease, Hip Osteonecrosis, Autologous implantation, Mesenchymal stromal cell, Humeral Osteonecrosis, Knee Osteonecrosis, Ankle Osteonecrosis
Brief summary
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow stem cells in sickle cell disease patients with osteonecrosis
Detailed description
The aim of this study is to evaluate autologous bone marrow stem cells transplantation as a safe and potentially beneficial treatment for sickle cell disease patients with osteonecrosis.
Interventions
PROCEDUREAutologous bone marrow stem cell graft
All patients will undergo autologous bone marrow stem cell implantation into the necrotic lesion area using a minimally invasive technique.
Sponsors
Hospital Universitário Professor Edgard Santos
Oswaldo Cruz Foundation
Federal University of Bahia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* Stable sickle cell disease patients * FICAT classification of osteonecrosis, inclusive of Stage II. * Diagnosis will be based on X-Ray and magnetic resonance imaging (MRI). * No infection in affected bones at the time of surgery. * Patient competent to give informed consent. * Scoring at least 20 points on the pain and daily life activities questionnaire.
Exclusion criteria
* FICAT stage III or more * Patients with a history of corticosteroids or on active therapy * Bone infection at the limb affected by necrosis * Recurrent painful crises, * Immunosuppressive drug therapy, * Pregnancy, * Presence of neoplastic disease or any other clinical concurrent condition other than sickle cell disease that predisposed them to the development of osteonecrosis of the femoral head
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Functional / Clinical Score of 10 points or more | 60 months | Standardized and objective scoring system to rate patient's functional abilities |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Numeric pain intensity scale (0-10) | 60 months | VAS scoring system |
| Disease progression defined as progression to a fractural stage of osteonecrosis | 60 months | Radiographic signals |
| Reoperation rate | 60 months | Need for further surgery or hip replacement |
| Radiological progression | 60 months | Radiological progression is defined according to the development of the Ficat- and Steinberg-staging system of osteonecrosis. It is based on radiological findings in X-Ray and MRI scans, respectively, and it is useful in predicting outcomes. |
Outcome results
None listed