OUTCOMES OF ALLOGENEIC HEMATOPOIETIC

STEM CELL TRANSPLANT FOR SAA AND PNH

AT VIETNAM NATIONAL INSTITUTE

OF HEMATOLOGY AND BLOOD TRANSFUSION

VO Thi Thanh Binh, MD

14-16 March, Korea

I have no personal or financial interests to declare:

I have no financial support from an industry source at the current presentation.

대한혈액학회 Korean Society of Hematology

COI disclosureName of author ： BINH, Vo Thi Thanh.

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SUBJECTS:

42 SAA and PNH patients underwent an allogeneic HSCT

at our institution from November 2010 to December 2018

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Conditioning regimen for non – malignant hematological diseases

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Enkhtsetseg Purev et al. Blood 2016;128:46895

3mg/kg, IV

Stem cell infusion

5mg/kg/day, oral in 12 months

Day

Transplant conditioning consisted of cyclophosphamide and fludarabine with horse ATG

MTX=methotrexate Cy=CyclophosphamideFlu=fludarabine ATH=Horse anti-thymocyte globulin 6

Characteristics of patients

undergoing allogeneic HSCT

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Characteristics No. of patients(N=42)

%

Median Age (Range): 23.7 ± 10.2 (5-42)

Diseases distribution SAA 32 80

PNH 7 12.5

SAA/PNH 3 7.5

Prior ATG/CSA treatmentYes 4 9.5

No 38 90.5

Stem cell sources

PBSC 37 90

BM/RCB or BM 2 5

URCB 2 5

Haplo-URCB 1

Types of allogeneic transplantMRD 39 92.5

URCB 2 5Haploidentical+/CBT 1 2.5

Conditioning regimen

Cy/Flu/+/-hATG 41 97.5

Cy post transplant 1 2.5

GVHD prophylaxis

CSA+ MTX 41 97.5

Tac/MMF 1 2.5

CD34+ dose on PBSC/BMSC 9.6 ± 3.1 (2.7-16) x10^6/kgSC dose on CB CD34: 2.9-7.9 x10^5/kg

TNC: 9.7-10 x10^7/kg Blood units transfused before transplantation 56.2 ± 42.1 (2-164) 8

RESULTS

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Neutrophil engraftment(Day)

PLT engraftment(Day)

21.0 ± 3.5 24.2 ± 4.0

Engraftment Status

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Engraftment No. of patients (%)

At Day+30 Yes 39 92.9

No 3 7.1

PBSC/BMSC or CBT

(n=40)

Yes 39 97.5

No 1 2.5

UCB alone (n=2)

Yes 0 0

No 2 100

Engraftment Data on 42 Patients

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Major complicationsNo. of

patients %

Infection Bacteria/fungalEarly 3 7.5

Late 3 7.5

CMV reactivation 14 30.3

GVHDAcute (I-II) 9 21.4

Chronic (mild/moderate) 13 30.9

Graft failurePrimary GF 2 4.8

Poor graft function 1 2.4

Graft rejection 3 7.1

Delay RBC/PLT recovery* 1 2.4

PRES 1 2.4

Complications

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3-year DFS and OS rates (n=42: ll Patients)

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3-year Disease Free Survival(N=42)

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3-year Overall Survival (N=42)

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Mortality after transplantation

Mortality No. patient (%)

100-day mortality after transplantation

3 7.1

One year mortality after transplantation

6 14.2

Causes of death: All from infection and graft failure.16

CASE REPORT:

UNRELATED CORD BLOOD AND HAPLOIDENTICAL

TRANSPLANT FOR SAA PATIENT AT NIHBT

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MEDICAL REPORT- Male 18 y.o. Diagnosis: Severe Aplastic Anemia

- Treated hATG/CSA: did not response

- Sister: 5/8 HLA matched

Plan to do URCBT plus haploidentical transplant after 6 months of IST.

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Combined Transplantation of UCB + CD34 Cells from a Haplo-matched Relative for SAA

Eligibility Criteria ANC < 500 cells/ul Failed to respond to immunosuppressive

therapy No HLA matched donor (related or

unrelated)

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RESULTS

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Neutrophil engraftment(Day)

PLT engraftment(Day)

D+11 D+15

Engraftment status

Blood cell counts at 10 months post transplant

WBC Hb PLT

6.4 G/L 140 g/L 260 G/L

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Chimerism status within 9 months

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The Cord-Haploidentical transplant patient transplant in May 2018

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CONCLUSION

At the Vietnam National Institute of Hematology and Blood transfusion:

Allogeneic HSCT from a matched sibling donor using Cy/Flu/ATG

was an effective and safe treatment for SAA and PNH patients: OS,

DFS at 3 years post transplant was 77.2% and 74% respectively.

Our preliminary result in one patient suggest that co-infusion of UCB

and haploidentical CD34+ cells is a feasible transplant option for SAA

patients who have failed IST and who lack an HLA matched donor.

Infection and graft failure were the most common complications

occurring post-transplant that led to transplant mortality.

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Success……

Happiness……

The 1st SAA patient transplant from MRD 2010

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The 100th transplant patient (2014)….The 1st pediatric SAA patient transplant from BM

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Co-operation with National Institute of Health in Washington DC (2008)

The 1st International Workshop on HSCT in emerging countries was hold in Hanoi, Vietnam November 2011

Acknowledgement

We deeply thank the experts:- From the National Institutes of Health in Washington DC- From Japanese Red Cross Nagoya First Hospital and

Aichi Cancer Center Research Institute.

We would like to thank the collaborators in VietnamNIHBT.

We also want to thank the patients who have confidencein our ability.

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