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. 2022 Jun 1;39(2):117-129.
doi: 10.4274/tjh.galenos.2022.2022.0084. Epub 2022 Apr 22.

Combination of Haploidentical Hematopoietic Stem Cell Transplantation with Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Severe Aplastic Anemia: A Retrospective Controlled Study

Xian-Fu Sheng  1 , Hui Li  1 , Li-Li Hong  1 , Haifeng Zhuang  1
Affiliations

Combination of Haploidentical Hematopoietic Stem Cell Transplantation with Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Severe Aplastic Anemia: A Retrospective Controlled Study

Xian-Fu Sheng et al. Turk J Haematol. .

Abstract
in English, Turkish

Objective: We retrospectively compared the outcomes of patients with severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (haplo-HSCT) combined or not combined with umbilical cord-derived mesenchymal stem cells (UC-MSCs).

Materials and methods: A total of 101 patients with SAA were enrolled in this study and treated with haplo-HSCT plus UC-MSC infusion (MSC group, n=47) or haplo-HSCT alone (non-MSC group, n=54).

Results: The median time to neutrophil engraftment in the MSC and non-MSC group was 11 (range: 8-19) and 12 (range: 8-23) days, respectively (p=0.049), with a respective cumulative incidence (CI) of 97.82% and 97.96% (p=0.101). Compared to the non-MSC group, the MSC group had a lower CI of chronic graft-versus-host disease (GVHD) (8.60±0.25% vs. 24.57±0.48%, p=0.048), but similar rates of grades II-IV acute GVHD (23.40±0.39% vs. 24.49±0.39%, p=0.849), grades III-IV acute GVHD (8.51±0.17% vs. 10.20±0.19%, p=0.765), and moderate-severe chronic GVHD (2.38±0.06% vs. 7.45±0.18%, p=0.352) were observed. The estimated 5-year overall survival (OS) rates were 78.3±6.1% and 70.1±6.3% (p=0.292) while the estimated 5-year GVHD-free, failure-free survival (GFFS) rates were 76.6±6.2% and 56.7±6.9% (p=0.045) in the MSC and non-MSC groups, respectively.

Conclusion: In multivariate analysis, graft failure was the only adverse predictor for OS. Meanwhile, graft failure, grades III-IV acute GVHD, and moderate-severe chronic GVHD could predict worse GFFS. Our results indicated that haplo-HSCT combined with UC-MSCs infusion was an effective and safe option for SAA patients.

Amaç: Göbek kordonundan elde edilen mezenkimal kök hücrelerin (UK-MKH’ler) infüzyonu ile kombine edilmiş ve edilmemiş, haploidentik hematopoietik kök hücre nakli (haplo-HKHN) yapılan şiddetli aplastik anemili (ŞAA) hastalarının sonuçları geriye dönük olarak karşılaştırıldı.

Gereç ve yöntemler: Bu çalışmaya ŞAA’lı toplam 101 hasta alındı ve hastalar haplo-HSCT ile birlikte UK-MKH infüzyonu (MKH grubu, N=47) ve tek başına haplo-HKHN (MKH olmayan grup, N=54) şeklinde iki gruba ayrıldı.

Bulgular: MKH alan ve almayan gruplarda nötrofil engraftmanı için medyan süreler sırasıyla, 11 (aralık, 8-19) ve 12 (aralık, 8-23) gün (p=0,049) ve kümülatif insidans (CI) sırası ile %97,82 ve %97,96 (p=0,101) bulundu. MKH almayan grupla karşılaştırıldığında, MKH alan grupta daha düşük kronik graft-versus-host hastalığı (cGVHD) CI saptandı (%8,60±0,25’e karşı %24,57±0,48, p=0,048), ancak II-IV akut GVHD (aGVHD) (%23,40±0,39-%24,49±0,39, p=0,849), III-IV aGVHD (%8,51±0,17 ve %10,20±0,19, p=0,765) ve orta-şiddetli cGVHD (%2,38±0,06 vs. %7,45±0,18, p=0,352) gruplarında fark görülmedi. MKH alan ve MKH almayan gruplarda tahmini beş yıllık genel sağkalım oranları %78,3±6,1 ve %70,1±6,3 (p=0,292) iken, GVHD’siz, hastalıksız sağkalım (GFFS) oranları sırasıyla, %76,6 ±%6,2 ve %56,7±6,9 (p=0,045) idi.

Sonuç: Çok değişkenli analizde greft yetmezliği, OS için tek olumsuz gösterge idi. Bu arada, greft yetmezliği, III-IV aGVHD ve orta-şiddetli cGVHD arasında daha kötü GFFS’yi öngörebilir. Sonuçlarımız, UC-MKH infüzyonu ile birlikte haplo-HKHN’nin ŞAA hastaları için etkili ve güvenli bir seçenek olduğunu gösterdi.

Keywords: Haploidentical; Severe aplastic anemia; Hematopoieticstem cell transplantation; Mesenchymal stem cells.

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Conflict of interest statement

Conflict of Interest: No conflict of interest was declared by the authors.

Figures

Figure 1
Figure 1
Cumulative incidences of graft-versus-host disease (GVHD) for the two groups. a) Cumulative incidences of grade II-IV acute GVHD (aGVHD): mesenchymal stem cell (MSC) group 23.40±0.39% vs. non-MSC group 24.49±0.39% (p=0.849). b) Cumulative incidences of grade III-IV aGVHD: MSC group 8.51±0.17% vs. non-MSC group 10.20±0.19% (p=0.765). c) Cumulative incidences of chronic GVHD (cGVHD): MSC group 8.60±0.25% vs. non-MSC group 24.57±0.48% (p=0.048). d) Cumulative incidences of moderate-severe cGVHD: MSC group 2.38±0.06% vs. non-MSC group 7.45±0.18% (p=0.352). HSCT: Hematopoietic stem cell transplantation.
Figure 2
Figure 2
Cumulative incidences of transplantation-related mortality (TRM) during follow-up in the mesenchymal stem cell (MSC) group and non-MSC group were 21.7±6.1% and 27.2±6.2%, respectively (p=0.424). HSCT: Hematopoietic stem cell transplantation.
Figure 3
Figure 3
Overall survival and graft-versus-host disease (GVHD)-free, failure-free survival of the two groups. a) Comparison of estimated 5-year overall survival between mesenchymal stem cell (MSC) group and non-MSC group (78.3±6.1% vs. 70.1±6.3%, p=0.292). b) Comparison of estimated 5-year GVHD-free, failure-free survival between MSC group and non-MSC group (76.6±6.2% vs. 56.7±6.9%, p=0.045). HSCT: Hematopoietic stem cell transplantation.
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