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Shinichiro Okamoto, MD, PhD
Asia-pacific Blood and Marrow Transplantation Group (APBMT)
Japan Society for Hematopoietic Cell Transplantation (JSHCT)
Design the future of HSCT – APBMT perspectives-
Years
1986 1989 1992 1995 1998 2001 2004 2007 2010 2013
5000
4000
2000
10000
9000
8000
7000
6000
3000
1000
Allogeneic HSCT
Autologous HSCT
Cumulative No. of HSCTs in APBMT Participating
Countries/Regions
Years
1986 1989 1992 1995 1998 2001 2004 2007 2010 2013
5000
4000
2000
6000
3000
1000
Related Donor HSCT
Unrelated Donor SHCT
Cumulative No. of Allogeneic HSCTs in
APBMT Participating Countries/Regions
Centers No. of HSCTs
Australia 40 1,509
China 58 3,402
Hong Kong 2 115
India 30 1,112
Iran 9 431
Japan 373 5,291
Korea 44 2,012
Malaysia - -
New Zealand 6 235
Pakistan 3 115
Philippines 2 7
Singapore 4 157
Thailand 7 213
Taiwan 18 477
Vietnam 7 47
Total 603 15,123
No. of Transplantations and Centers in 2013
Participating in Activity Survey 2013
0
1000
2000
3000
4000
Allogeneic (Total N=9,332) Autologous (Total N=5,791)
Indications for Hematopoietic Stem Cell Transplants in 2013
-APBMT Activity Survey-
NumberofTransplants
Courtesy of Dr. Minako Iida
• There are also many challenges…...
APBMT: Current status
• Given the size of the population we serve and the
spectrum of disease we see in this region. There are
many opportunities to explore the way to improve the
current approach of HSCT
• Our group consists of countries/regions where the
indication, infrastructure, financial background,
environment (ex. endemic infectious diseases) for HSCT
vary significantly.
• Unmet need of but limited access to HSCT (and non-
transplant novel therapies)
• Limited training opportunities for HSCT in Asia-Pacific
countries/regions.
• Difficulties for developing infrastructure for HSCT
while ensuring standards.
APBMT: Major Challenges facing HSCT(1)
APBMT: Addressing Challenges (1)
Education & Training
• Understand the needs in each emerging country.
• Survey on training opportunities in HSCT in Asia Pacific
countries/regions, and make the information available
our web site.
• Prepare standardized educational program for MD and
other professionals (Nurse, Pharmacist, HCTC).
• Focus on the candidates who are expected to lead the
next generation of HSCT in each country/region.
• Educational session at APBMT Annual Meeting an make
the sessions PP/video available at web site.
APBMT: Addressing Challenges (1) cont’d
Infrastructure & standards
• Prepare standardized approach for setting up transplant
facility.
• Harmonize our approaches with the materials and
recommendations of WBMT.
Collaboration
- Promote collaborative study/research among Asia
Pacific countries/regions to make evidences.
- Steer the HSCT Transplant Outcome Registry in Asia
pacific countries/regions (APBMT Registry) and
to set-up platform to design and promote clinical study
APBMT: Addressing Challenges (1) cont’d
Potential barriers to collaborate
 Regulatory requirements/burdensome review process
 Insurance coverage for patients
 Inadequate academic credit/carrier disincentive
 Fragmented infrastructure/incompatible data base
 Lack of qualified investigators and other personnel
 Lack of willing participants to trials
New Hope
Matched Unrelated Donor
transplant
Haplo strikes back!
HLAmismatched
(haploidentical) transplant
Haploidentical HSCT in APBMT
Courtesy of Dr. M. Iida
3000
2500
2000
1500
1000
500
2009 201420122008 201320112006 20102007
3000
2500
2000
1500
1000
500
2009 201420122008 201320112006 20102007
Haplo-BM
Haplo-PB(Including PB+BM)
BM vs PB
1200
1000
800
600
400
200
2009 201420122008 201320112006 20102007
BM + PB(Except for China)
• Field shifting toward HSCT being done in elderly patients with
more comorbidities.
• Increasing number of HSCT survivors requiring long term care.
• Workforce shortages are expected in transplant physicians,
nurses, and other allied healthcare workers.
• The quality and value measure for optimizing the use of HSCT
in elderly patients.
• Current inability of the primary care workforce to absorb
caring for post-transplant patients.
• Introduction of non-transplant novel therapies.
APBMT: Major Challenges facing HSCT (2)
21
Proportion of elderly population(age 65 ≤)
is rapidly growing in Japan
45
10
35
20
40
5
25
15
30
%
China
India
Indonesia
Japan
Philippine
Korea
Singapore
Thailand
Patiens
Team C Community resource
JSHCT, MHLW (Government)
Designated local hub hospital promoting HSCT
Team A
Active care team
Team B
Best support team
Team L
LTFU
Team H
Home Care
Fuji S, presented at JSH 2014
Solid lines: predicted Dotted lines: observed
0.80
0.40
0.20
0.60
1.00
400200 600 800
79%
Probability
Time after SCT (days)
64%
42%
27%
HCT-CI, HCT-CI-Age, PAM score EBMT score
Score including HCT-CI, Age, PS, Disease risk, and HLA
disparity
Score System predicting transplant outcomes
Life with good
quality of life
Cost for HSCT
Cost for late
complications
Limited recourses
of our society
Survival is no longer sole measure of benefit for elderly
undergoing HSCT
GVHD-free, relapse-free survival (GRFS)
Composite end point of transplant outcomes after Allo-HSCT
GRFS is defined as the absence of
- Grade Ⅲ-Ⅳ acute GVHD
- Systemically treated chronic GVHD
- Relapse
- Death
GRFS is a patient-centered definition
of success that represents ideal
recovery without significant GVHD-
related morbidity.
GVHD
GVL
Comparison of Graft-versus-Host Disease-free, Relapse-
free Survival according to a Variety of Graft Sources
Inamoto Y for JSHCT GVHD WG Hematologica 2016 E-pub ahead
-Patients aged 20< with hematological malignancies (Standard risk)
CumulativeIncidenceofFailure
0.8
0.4
0.2
0.6
1.0
3 186 12 2415 219
Age≥21
5/6 SIB BM
8/8 UR BM
6-7/8 UR BM
6/6 SIB PB
6/6 SIB BM
UR CB
5/6 SIB PB
0 40 10060 8020
(%)
Death Relapse Ⅲ-Ⅳ Acute GVHD Chronic GVHD
Months after Transplantation
AdjustedGRFS
0.8
0.4
0.2
0.6
1.0
3 186 12 2415 219
Months after Transplantation
6/6 SIB PB
8/8 UR BM
6/8 UR BM
5/6 SIB BM
6/6 SIB BM
7/8 UR BM
5/6 SIB PB
CB
Relapse
Death
Chronic GVHD
Ⅲ-Ⅳ Acute GVHD
This is the future of HSCT in 23rd Centuries
HSCT?
What is that, the Dark Ages?
Here , swallow this.
If you have problem, call me
The doctor gave me a pill and
I grew a new bone marrow

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Shinichiro Okamoto - Design the Future of HACT: APBMT Perspectives

  • 1. Shinichiro Okamoto, MD, PhD Asia-pacific Blood and Marrow Transplantation Group (APBMT) Japan Society for Hematopoietic Cell Transplantation (JSHCT) Design the future of HSCT – APBMT perspectives-
  • 2.
  • 3. Years 1986 1989 1992 1995 1998 2001 2004 2007 2010 2013 5000 4000 2000 10000 9000 8000 7000 6000 3000 1000 Allogeneic HSCT Autologous HSCT Cumulative No. of HSCTs in APBMT Participating Countries/Regions
  • 4. Years 1986 1989 1992 1995 1998 2001 2004 2007 2010 2013 5000 4000 2000 6000 3000 1000 Related Donor HSCT Unrelated Donor SHCT Cumulative No. of Allogeneic HSCTs in APBMT Participating Countries/Regions
  • 5. Centers No. of HSCTs Australia 40 1,509 China 58 3,402 Hong Kong 2 115 India 30 1,112 Iran 9 431 Japan 373 5,291 Korea 44 2,012 Malaysia - - New Zealand 6 235 Pakistan 3 115 Philippines 2 7 Singapore 4 157 Thailand 7 213 Taiwan 18 477 Vietnam 7 47 Total 603 15,123 No. of Transplantations and Centers in 2013 Participating in Activity Survey 2013
  • 6.
  • 7. 0 1000 2000 3000 4000 Allogeneic (Total N=9,332) Autologous (Total N=5,791) Indications for Hematopoietic Stem Cell Transplants in 2013 -APBMT Activity Survey- NumberofTransplants Courtesy of Dr. Minako Iida
  • 8.
  • 9. • There are also many challenges…... APBMT: Current status • Given the size of the population we serve and the spectrum of disease we see in this region. There are many opportunities to explore the way to improve the current approach of HSCT • Our group consists of countries/regions where the indication, infrastructure, financial background, environment (ex. endemic infectious diseases) for HSCT vary significantly.
  • 10. • Unmet need of but limited access to HSCT (and non- transplant novel therapies) • Limited training opportunities for HSCT in Asia-Pacific countries/regions. • Difficulties for developing infrastructure for HSCT while ensuring standards. APBMT: Major Challenges facing HSCT(1)
  • 11. APBMT: Addressing Challenges (1) Education & Training • Understand the needs in each emerging country. • Survey on training opportunities in HSCT in Asia Pacific countries/regions, and make the information available our web site. • Prepare standardized educational program for MD and other professionals (Nurse, Pharmacist, HCTC). • Focus on the candidates who are expected to lead the next generation of HSCT in each country/region. • Educational session at APBMT Annual Meeting an make the sessions PP/video available at web site.
  • 12. APBMT: Addressing Challenges (1) cont’d Infrastructure & standards • Prepare standardized approach for setting up transplant facility. • Harmonize our approaches with the materials and recommendations of WBMT.
  • 13. Collaboration - Promote collaborative study/research among Asia Pacific countries/regions to make evidences. - Steer the HSCT Transplant Outcome Registry in Asia pacific countries/regions (APBMT Registry) and to set-up platform to design and promote clinical study APBMT: Addressing Challenges (1) cont’d
  • 14. Potential barriers to collaborate  Regulatory requirements/burdensome review process  Insurance coverage for patients  Inadequate academic credit/carrier disincentive  Fragmented infrastructure/incompatible data base  Lack of qualified investigators and other personnel  Lack of willing participants to trials
  • 15.
  • 16.
  • 17. New Hope Matched Unrelated Donor transplant Haplo strikes back! HLAmismatched (haploidentical) transplant
  • 18. Haploidentical HSCT in APBMT Courtesy of Dr. M. Iida 3000 2500 2000 1500 1000 500 2009 201420122008 201320112006 20102007 3000 2500 2000 1500 1000 500 2009 201420122008 201320112006 20102007 Haplo-BM Haplo-PB(Including PB+BM) BM vs PB 1200 1000 800 600 400 200 2009 201420122008 201320112006 20102007 BM + PB(Except for China)
  • 19.
  • 20. • Field shifting toward HSCT being done in elderly patients with more comorbidities. • Increasing number of HSCT survivors requiring long term care. • Workforce shortages are expected in transplant physicians, nurses, and other allied healthcare workers. • The quality and value measure for optimizing the use of HSCT in elderly patients. • Current inability of the primary care workforce to absorb caring for post-transplant patients. • Introduction of non-transplant novel therapies. APBMT: Major Challenges facing HSCT (2)
  • 21. 21 Proportion of elderly population(age 65 ≤) is rapidly growing in Japan 45 10 35 20 40 5 25 15 30 % China India Indonesia Japan Philippine Korea Singapore Thailand
  • 22. Patiens Team C Community resource JSHCT, MHLW (Government) Designated local hub hospital promoting HSCT Team A Active care team Team B Best support team Team L LTFU Team H Home Care
  • 23. Fuji S, presented at JSH 2014 Solid lines: predicted Dotted lines: observed 0.80 0.40 0.20 0.60 1.00 400200 600 800 79% Probability Time after SCT (days) 64% 42% 27% HCT-CI, HCT-CI-Age, PAM score EBMT score Score including HCT-CI, Age, PS, Disease risk, and HLA disparity Score System predicting transplant outcomes
  • 24. Life with good quality of life Cost for HSCT Cost for late complications Limited recourses of our society Survival is no longer sole measure of benefit for elderly undergoing HSCT
  • 25. GVHD-free, relapse-free survival (GRFS) Composite end point of transplant outcomes after Allo-HSCT GRFS is defined as the absence of - Grade Ⅲ-Ⅳ acute GVHD - Systemically treated chronic GVHD - Relapse - Death GRFS is a patient-centered definition of success that represents ideal recovery without significant GVHD- related morbidity. GVHD GVL
  • 26. Comparison of Graft-versus-Host Disease-free, Relapse- free Survival according to a Variety of Graft Sources Inamoto Y for JSHCT GVHD WG Hematologica 2016 E-pub ahead -Patients aged 20< with hematological malignancies (Standard risk) CumulativeIncidenceofFailure 0.8 0.4 0.2 0.6 1.0 3 186 12 2415 219 Age≥21 5/6 SIB BM 8/8 UR BM 6-7/8 UR BM 6/6 SIB PB 6/6 SIB BM UR CB 5/6 SIB PB 0 40 10060 8020 (%) Death Relapse Ⅲ-Ⅳ Acute GVHD Chronic GVHD Months after Transplantation AdjustedGRFS 0.8 0.4 0.2 0.6 1.0 3 186 12 2415 219 Months after Transplantation 6/6 SIB PB 8/8 UR BM 6/8 UR BM 5/6 SIB BM 6/6 SIB BM 7/8 UR BM 5/6 SIB PB CB Relapse Death Chronic GVHD Ⅲ-Ⅳ Acute GVHD
  • 27. This is the future of HSCT in 23rd Centuries HSCT? What is that, the Dark Ages? Here , swallow this. If you have problem, call me The doctor gave me a pill and I grew a new bone marrow

Editor's Notes

  1. Slide 12: The most common indications for HCT in the US in 2013 were multiple myeloma and lymphoma, accounting for 52% of all HCTs. AML and myelodysplasia are the most common indications for allogeneic transplants accounting for 53% of allogeneic HCTs.
  2. Deserve Particular attention