Abstract Number: OC 49.3
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Hemophilic arthropathy is a serious clinical complication in patients with severe hemophilia. Mononuclear stem cells(MNSC) are pluripotent cells with the ability to be differentiated to specialized adult cells, such as chondrocytes.
Aims: To evaluate the effectiveness and safety of intraarticular injection of MNSC in hemophilia patients with arthropathy.
Methods: Fifty to seventy cc bone marrow was taken from each patient. A minimum of 40 million autologous MNSCs were extracted and injected to the target joint. Hemophilia Joint Health Score(HJHS), Pettersson(PS), Arnold Hilgartner(AH) and Denver magnetic resonance imaging (DMRI) scores were applied at baseline, 3 ,6 and 12 months after injection, to evaluate the degree of changes in joint damage.
Results: Four patients were studied. No significant progress of joint distruction had been found during follow up for all patients except patient 1 who had bleeding (Table 1).Patient 1’s AH score showed no change during the study but HJHS,PS and DMRI scores increased. the patient had bleeding between 6-12 month follow up.The patinet 2 and patients 3 (ankle joint) had no significant change.The patient 3 (elbow joint) had trivial improvement at HJHS. Other scores were stable. Patient 4 had an accident at 11th month of follow up that resulted in knee fraction and major bleeding .No side effects were observed in all patients.
| Pettersson score(PS) 0-13 points higher score is worse |
Arnold-Hilgartner score(AH) I-V points higher score is worse |
Denver magnetic resonance imaging score(DMRI) 0-10 points higher score is worse |
Hemophilia joint health score (HJHS) max=124 points higher score is worse |
|
| Patient 1, Right knee |
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| Baseline 3 month 6 month 12 month |
2 3 2 4 |
I II I II |
9 9 9 10 |
7 – 8 12 |
| Patient 2, Right knee |
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| Baseline 3 month 6month 12 month |
5 6 7 7 |
IV IV IV IV |
9 10 10 10 |
15 7 7 – |
| Patient 3, Right elbow |
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| Baseline 3 month 6 month 12 month |
5 5 5 5 |
IV IV IV IV |
10 10 10 10 |
14 – 11 7 |
| Patient 3, Ankle |
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| Baseline 3 month 12 month |
6 6 7 |
IV IV IV |
10 10 10 |
5 6 7 |
| Patient 4, Right knee |
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| Baseline 3 month 6 month 12 month |
7 6 5 NA |
IV IV IV NA |
10 10 10 NA |
12 10 11 NA |
The baseline, 3 ,6 and 12 months post-procedure finding for the joints studied
Conclusions: The 6-month follow-up showed improvement in comparison to 3-month follow-up. Worsening score at 3 months’ follow-up compared to the baseline can be interpreted by arthritis or mild bleeding after intra articular injection of MNSC. The 12-month follow up showed no progress in joint damage in comparison to the base line in all patients except one patient who complicated with one bleeding episode that led to decrease the efficacy of improvement. Our results indicated the possible protective effect and safety of MNSCs in progression of hemophilic arthropathy.
To cite this abstract in AMA style:
Karimi M, Shahsavani A, Ayatollahi M, Haghpanah S, Tahami M, Iranpour P, Bordbar M, Tavoosi H, Azizkhani S, Amirmoezi F. Efficacy and Safety of Intraarticular Injection of Mononuclear Stem Cells in Hemophilia Patients with Arthropathy [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/efficacy-and-safety-of-intraarticular-injection-of-mononuclear-stem-cells-in-hemophilia-patients-with-arthropathy/. Accessed November 29, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-and-safety-of-intraarticular-injection-of-mononuclear-stem-cells-in-hemophilia-patients-with-arthropathy/