Table 3.

Summary of features and results of clinical studies applying expanded ASCs for knee OA treatment

References	Cell donor	Patients			Study design				Study results
		Number of patients	Knee OA grading	Age range	Type of study (ClinicalTrials.gov Identifier if applies)	Experimental group(s)	Cell dosage	Treatment comparator	Final follow-up	Measurements	Main outcomes
Jo [35]	Autologous	Phase I: 9 (3/group) Phase II: 9	KL II–IV	18–75 years	Proof-of-concept clinical trial	Phase I: injection with three different ASC doses Phase II injection with the highest ASC dose	Phase I: 10 × 106 ASCs 50 × 106 ASCs 100 × 106 ASCs Phase II: 100 × 106 ASCs	–	6 months	Safety VAS WOMAC OA index Second-look arthroscopy Histology MRI	Treatment safe for all the tested ASC doses ↓ Pain and ↑ knee function only in the high-dose group
Pers [36]	Autologous	18 (6/group)	KL III–IV	50–75 years	Phase I multicentric, prospective, single-arm, open-label, dose escalating clinical trial (NCT01585857)	Injection with three different ASC doses	2 × 106 ASCs 10 × 106 ASCs 50 × 106 ASCs	–	6 months	VAS WOMAC OA index Patient global assessment Knee injury and OA outcome score Short arthritis assessment scale SF-36 quality-of-life questionnaire	↓ Pain and ↑ knee function only in the low-dose group
Jo [37]	Autologous	Phase I: 9 (3/group) Phase II: 9	KL II–IV	18–75 years	2-year follow-up of the trial described in [35]	Phase I: injection with three different ASC doses Phase II injection with the highest ASC dose	Phase I: 10 × 106 ASCs 50 × 106 ASCs 100 × 106 ASCs Phase II: 100 × 106 ASCs	–	24 months	VAS WOMAC OA index Knee society clinical rating system Knee injury and OA outcome score MRI	At 1 year, significant improvements mainly in the high-dose group Only in the high-dose group, improvements maintained at 2 years
Song [38]	Autologous	Phase I: 18 (6/group) Phase II: 14 (the same patients treated in Phase I)	KL II–IV	40–70 years	Phase I/II randomized, double-blind clinical trial (NCT01809769)	Injection with three different ASC doses. Two injections at 3 and 6 weeks after liposuction A third ASC injection was provided after 48 weeks	Phase I: 10 × 106 ASCs 20 × 106 ASCs 50 × 106 ASCs Phase II: third injection with 50 × 106 ASCs	–	24 months	WOMAC OA index Numerical pain rating scale SF-36 quality-of-life questionnaire MRI	Treatment safe for all the tested ASC doses ↓ Pain, ↑ knee function, ↑ cartilage volume more relevant and long-lasting in the high-dose group The third injection increased the improvement rate, especially in patients previously treated with the low and middle ASC dose