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Can All Thalassemia Patients Be Cured 
with HSCT? 
Suradej Hongeng, MD 
Ramathibodi Hospital, Mahidol University
Pathophysiology of b-Thalassemia/Hb E Disease
Treatments for Severe Thalassemia 
Palliative treatment 
Blood transfusion 
Iron chelation 
Splenectomy 
Curative treatment 
Hematopoietic stem cell transplant (HSCT) 
Allogeneic HSC 
Gene therapy 
Autologous HSC
BMT in Thalassemia and Lucarelli Classification 
Risk factors for BMT in thalassemia 
Chelation Regular vs Irregular 
Hepatomegaly Absent vs Present 
Liver fibrosis Absent vs Present 
Risk classes for BMT in thalassemia 
Chelation Hepatomegaly Fibrosis 
Class1 Regular NO NO 
Class2 Reg/Irreg NO/YES NO/YES 
Class3 Irregular YES YES 
Lucarelli G et al. N Engl J Med 1990
Sabloff et al. ICBMTR, Blood 
2011
Results of MRD in HSCT for Thal Patients 
Reference Patients OS TFS 
Di Bartolomeo et al. 111 90 86 
Argiolu et al. 37 88 88 
Clift et al. 68 94 81 
Lawson et al. 54 95 82 
Ghavamzadehv et al. 60 83 73 
Denninson et al. 50 76 68 
Lin et al. 28 86 82 
Lee et al. 44 86 82 
Issaragrisil et al. 21 70 53
HSCT in Thalassemia at Ramathibodi 
Related n=28, Unrelated n=21 Total 49 patients 
82% 
71% 
Overall survival (OS) and thalassemia free survival (TFS) in Thai children 
Hongeng S et al. Biol Blood Marrow Transplant 2006 
92% 
82%
Dilemma of HSCT in Thal 
Donor availability 
Older patients
Number of Donors in 
Registry 
Percentage of 
patients with 
45% 
40% 
35% 
30% 
Donors Percent of Finding 
6% 5% 6% 7% 
24% 
18% 
18% 
Percent of Finding 
45% 
25% 
20% 
15% 
10% 
5% 
HLA-A,-B,-DR Matched Donor 
HLA-A,-B,-DR Matched Donor 
within 1 month 
within 1 month 
37% 
0% 
200320042005200620072008200920102011
New Stratification for 
High Risk Class 3 Patients 
Definition 
Older patients 
Age > 10 yrs 
Hepatomegaly (liver > 3 cm below right 
costal margin)
High Risk Class 3 Patients 
(Age > 10 yrs) 
Pretransplant Management Program 
Hypertransfusion in order to decrease erythroid 
expansion especially to decrease spleen size 
Regular iron chelation for at least 6-12 months 
Hydroxyurea (Hb F enhancer) in order to decrease 
erythroid expansion: 20 mg/kg/day for at least 
6-12 months 
Sodani P et al. Blood 2004 
Hongeng S et al. Am J Hematol 2007
Previous RTC Regimen 
(Early 8 Patients) 
Busulfan oral (8-12 mg/kg) 
Fludarabine (210 mg/m2) 
ATG (Fresinius 20 mg/kg) 
+TLI 500 cGy 
+ Thiotepa 10 mg/kg 
+ Melphalan 100 mg/m2 
GVHD prophylaxis
Am J Hematol, 2007 
2 Iannone R, et al. BBMT 2003 
3 Horan JT, et al. BMT 2005 
4 Jacobsohn DA, et al. Lancet 2004 
5 Krishnamurti L, et al. BMT 2006
Outcomes of Thalassemia Patients Undergoing 
Hematopoietic Stem Cell Transplant by Using 
a Standard Myeloablative (MAC) Versus 
a Novel Reduced Toxicity (RTC) Conditioning 
Regimen According to a New Risk Stratification 
Suradej Hongeng, MD 
Dept of Pediatrics, Faculty of Medicine 
Ramathibodi Hospital, Mahidol University 
Bangkok, Thailand
Novel Approach for High Risk Class 3 Patients 
Pre transplant management 
Hypertransfusion, chelation and hydroxyurea 
Pretransplant immunosuppression (PTIS) 
Flu + Dex (2 cycles) 
Conditioning regimen 
Bu + Flu + ATG
Pre-transplant Immunosuppression (PTIS) 
Fludarabine 40 mg/m2 x 5 days 
Dexamethasone 25 mg/m2 x 5 days 
1-2 cycles; 28-day cycle 
PTIS is given prior to conditioning regimen
Decreased CD4 cell proliferation
Conditioning Regimen for 
High Risk Class 3 Patients 
Past 
Combination of cyclophosphamide & busulfan 
Too much alkylating agent regimen 
Too toxic
Novel RTC Regimen and GVHD Prophylaxis 
Fludarabine 35 mg/m2; d-9,-8,-7,-6,-5,-4 
Busulfex 130 mg/m2; d-9,-8,-7,-6 
ATG (Thymoglobulin) 1.5 mg/kg; d-3,-2,-1 
CSA or FK506 and MMF (60 days)
18 high risk 
class 3 patients 
BBMT;2013
MAC vs Novel RTC
MAC Regimens and GVHD Prophylaxis 
Related donor and age < 10 yrs 
MAC regimen: Cyclo 200 mg/kg, Bu 14-16 mg/kg PO/IV 
CSA + MTX 
Unrelated group and < 10 yrs 
MAC regimen: Cyclo 200 mg/kg, Bu 14-16 mg/kg PO/IV, 
Flu 210 mg/m2 and ATG (Fresenius) 40 mg/kg 
FK506 + MTX
HLA Matching 
Related: 6 allele matching 
Unrelated: (before 2006) 6 allele matching 
(current) 8 allele matching
Study Population 
120 thalassemia patients undergoing HSCT; 1989 - 2013 
Exclude cord blood transplant n = 7 
Exclude previous RTC transplant n = 8 
(Am J Hematol; 2007) 
Exclude haploidentical transplant n = 7 
Final number of patients n = 98 patients 
Related n = 65; Unrelated n = 33 
MAC n = 76; Novel RTC n = 22
Patient Characteristics 
MAC 
Related n= 50 , Unrelated n=26 (34%) 
Mismatched HLA (1 Ag or 1 Allele) 12/76 (15%) 
Novel RTC 
Related n=15 , Unrelated n=7 (32%) 
Mismatched HLA (1 Ag or 1 Allele) 5/22 (22%) 
All received BM or PBSC.
Novel RTC Group 
22 patients 
2 out of 22 had second HSCT and 1 had third HSCT 
Age; median = 16 (10-21) y/o 
Male 8; Female 14 
Splenectomy = 7 (Referral hospital) 
All patients had liver > 5 cm below costal margin. 
Ferritin level; median = 3100 (869-8350) ng/mL 
Comorbidities 
2 DM, 1 previous extramedullary hemopoiesis, 
1 previous history of PHT
Novel RTC Group 
21 patients received PBSC. 
1 patient received BM. 
Median CD34+ 9.4 (4.67-19.26) x106 cells/kg 
MRD 13 MMRD 2 (DRB1) 
MUD 4 MMUD 2 (C) MMUD 1 (A)
MAC vs RTC
Outcome of All 98 Patients 
Overall survival = 94% (95%CI; 86.3%-97/1%) 
Event free survival = 87% (95%CI; 76.6%-91.1%)
Survival Rates Related (n=65) and 
Unrelated (n=33) HSCT in Thalassemias 
OS Related vs Unrelated = 94% 
EFS Related = 88% 
EFS Unrelated = 82%
MAC (n = 76) vs Novel RTC (n= 22) 
OS MAC = 95% 
RTC = 90% 
EFS MAC = 88% 
RTC = 93%
MAC vs Novel RTC 
MAC RTC 
Acute GVHD gr III-IV 3 (4%) 2 (10%) 
Chronic GVHD 
Limited 6 (8%) 3 (13%) 
Extensive 2 (3%) 0 
Dead 3 2 
1 Graft failure 1 Fungal infection 
1 Viral infection 1 Accident 
1 Secondary AML
Haploidentical HSCT (34+ selection) 
22 pts 
Myeloablative 
regimen; Cyclo, Bu, 
Flu,Thiotepa and 
ATG 
GVHD prophylaxis 
CSA 
Sodani et al, Blood 2010
All Thalassemia Patients May Be Cured with HSCT? 
TFS in related or unrelated for all age group 
(including pts age older than 10 yrs) in our center is 90%. 
Haploindentical HSCT in 12 severe patients 
(9/12 age > 10 yr) 
RTC plus Cyclophosphamide post transplant 
All 12 pts survived without thalassemia. 
No major complication except GvHD gr I-II. 
Follow up time; 4-20 months 
(abstract submitted to ASH 2014)
Gene Therapy
Overview of the clinical protocol 
Testing 
and Release 
While Frozen 
Maximize 
% Transduced 
HSCs 
Vector 
+ 
Cytokines 
Bone Marrow 
Harvest 
IV Infusion 
Transduced Cells 
(> 4x106 CD34+/Kg) 
(Spontaneous Homing) 
CD34+ cells 
(2x108 unsorted BM cells/Kg 
kept for rescue)
Conclusion 
Outcomes of HSCT for thal in both MRD and MUD are 
favorable. 
Outcomes of HSCT for all age group among thal pts are 
also favorable. 
Whether the novel RTC regimen should be studied in 
younger thalassemia patients is needed to be 
investigated. 
Autologous HSC with gene addition may give a new 
hope for cure for thal patients.
Acknowledgement 
Samart Pakakasama 
Ampaiwan Chuansumrit 
Nongnuch Sirachainan 
Usanarat Anurathapan 
Duantida Songdej 
HLA lab and BMT nurses 
Somtawain Sirirueng 
Wanpen Pantangkool 
Saengsuree Jootar 
Artit Ungkanont 
Vinai Suvattee 
Suthat Fucharoen 
Surapol Issaragrisil 
Borje Andersson 
Pramongkutklao Hospital 
Srinakarind Hospital 
Suandok Hospital 
Songklanakarin Hospital 
Siriraj Hospital
Acknowledgement 
Ramathibodi Foundation 
Children Cancer Fund under The 
Patronage of HRH Princess Som Sawali

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Outcomes of HSCT for Thalassemia Patients Using MAC vs Novel RTC Regimens

  • 1. Can All Thalassemia Patients Be Cured with HSCT? Suradej Hongeng, MD Ramathibodi Hospital, Mahidol University
  • 3. Treatments for Severe Thalassemia Palliative treatment Blood transfusion Iron chelation Splenectomy Curative treatment Hematopoietic stem cell transplant (HSCT) Allogeneic HSC Gene therapy Autologous HSC
  • 4. BMT in Thalassemia and Lucarelli Classification Risk factors for BMT in thalassemia Chelation Regular vs Irregular Hepatomegaly Absent vs Present Liver fibrosis Absent vs Present Risk classes for BMT in thalassemia Chelation Hepatomegaly Fibrosis Class1 Regular NO NO Class2 Reg/Irreg NO/YES NO/YES Class3 Irregular YES YES Lucarelli G et al. N Engl J Med 1990
  • 5. Sabloff et al. ICBMTR, Blood 2011
  • 6. Results of MRD in HSCT for Thal Patients Reference Patients OS TFS Di Bartolomeo et al. 111 90 86 Argiolu et al. 37 88 88 Clift et al. 68 94 81 Lawson et al. 54 95 82 Ghavamzadehv et al. 60 83 73 Denninson et al. 50 76 68 Lin et al. 28 86 82 Lee et al. 44 86 82 Issaragrisil et al. 21 70 53
  • 7. HSCT in Thalassemia at Ramathibodi Related n=28, Unrelated n=21 Total 49 patients 82% 71% Overall survival (OS) and thalassemia free survival (TFS) in Thai children Hongeng S et al. Biol Blood Marrow Transplant 2006 92% 82%
  • 8. Dilemma of HSCT in Thal Donor availability Older patients
  • 9. Number of Donors in Registry Percentage of patients with 45% 40% 35% 30% Donors Percent of Finding 6% 5% 6% 7% 24% 18% 18% Percent of Finding 45% 25% 20% 15% 10% 5% HLA-A,-B,-DR Matched Donor HLA-A,-B,-DR Matched Donor within 1 month within 1 month 37% 0% 200320042005200620072008200920102011
  • 10. New Stratification for High Risk Class 3 Patients Definition Older patients Age > 10 yrs Hepatomegaly (liver > 3 cm below right costal margin)
  • 11. High Risk Class 3 Patients (Age > 10 yrs) Pretransplant Management Program Hypertransfusion in order to decrease erythroid expansion especially to decrease spleen size Regular iron chelation for at least 6-12 months Hydroxyurea (Hb F enhancer) in order to decrease erythroid expansion: 20 mg/kg/day for at least 6-12 months Sodani P et al. Blood 2004 Hongeng S et al. Am J Hematol 2007
  • 12. Previous RTC Regimen (Early 8 Patients) Busulfan oral (8-12 mg/kg) Fludarabine (210 mg/m2) ATG (Fresinius 20 mg/kg) +TLI 500 cGy + Thiotepa 10 mg/kg + Melphalan 100 mg/m2 GVHD prophylaxis
  • 13. Am J Hematol, 2007 2 Iannone R, et al. BBMT 2003 3 Horan JT, et al. BMT 2005 4 Jacobsohn DA, et al. Lancet 2004 5 Krishnamurti L, et al. BMT 2006
  • 14. Outcomes of Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplant by Using a Standard Myeloablative (MAC) Versus a Novel Reduced Toxicity (RTC) Conditioning Regimen According to a New Risk Stratification Suradej Hongeng, MD Dept of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University Bangkok, Thailand
  • 15. Novel Approach for High Risk Class 3 Patients Pre transplant management Hypertransfusion, chelation and hydroxyurea Pretransplant immunosuppression (PTIS) Flu + Dex (2 cycles) Conditioning regimen Bu + Flu + ATG
  • 16. Pre-transplant Immunosuppression (PTIS) Fludarabine 40 mg/m2 x 5 days Dexamethasone 25 mg/m2 x 5 days 1-2 cycles; 28-day cycle PTIS is given prior to conditioning regimen
  • 17. Decreased CD4 cell proliferation
  • 18. Conditioning Regimen for High Risk Class 3 Patients Past Combination of cyclophosphamide & busulfan Too much alkylating agent regimen Too toxic
  • 19. Novel RTC Regimen and GVHD Prophylaxis Fludarabine 35 mg/m2; d-9,-8,-7,-6,-5,-4 Busulfex 130 mg/m2; d-9,-8,-7,-6 ATG (Thymoglobulin) 1.5 mg/kg; d-3,-2,-1 CSA or FK506 and MMF (60 days)
  • 20. 18 high risk class 3 patients BBMT;2013
  • 22. MAC Regimens and GVHD Prophylaxis Related donor and age < 10 yrs MAC regimen: Cyclo 200 mg/kg, Bu 14-16 mg/kg PO/IV CSA + MTX Unrelated group and < 10 yrs MAC regimen: Cyclo 200 mg/kg, Bu 14-16 mg/kg PO/IV, Flu 210 mg/m2 and ATG (Fresenius) 40 mg/kg FK506 + MTX
  • 23. HLA Matching Related: 6 allele matching Unrelated: (before 2006) 6 allele matching (current) 8 allele matching
  • 24. Study Population 120 thalassemia patients undergoing HSCT; 1989 - 2013 Exclude cord blood transplant n = 7 Exclude previous RTC transplant n = 8 (Am J Hematol; 2007) Exclude haploidentical transplant n = 7 Final number of patients n = 98 patients Related n = 65; Unrelated n = 33 MAC n = 76; Novel RTC n = 22
  • 25. Patient Characteristics MAC Related n= 50 , Unrelated n=26 (34%) Mismatched HLA (1 Ag or 1 Allele) 12/76 (15%) Novel RTC Related n=15 , Unrelated n=7 (32%) Mismatched HLA (1 Ag or 1 Allele) 5/22 (22%) All received BM or PBSC.
  • 26. Novel RTC Group 22 patients 2 out of 22 had second HSCT and 1 had third HSCT Age; median = 16 (10-21) y/o Male 8; Female 14 Splenectomy = 7 (Referral hospital) All patients had liver > 5 cm below costal margin. Ferritin level; median = 3100 (869-8350) ng/mL Comorbidities 2 DM, 1 previous extramedullary hemopoiesis, 1 previous history of PHT
  • 27. Novel RTC Group 21 patients received PBSC. 1 patient received BM. Median CD34+ 9.4 (4.67-19.26) x106 cells/kg MRD 13 MMRD 2 (DRB1) MUD 4 MMUD 2 (C) MMUD 1 (A)
  • 29. Outcome of All 98 Patients Overall survival = 94% (95%CI; 86.3%-97/1%) Event free survival = 87% (95%CI; 76.6%-91.1%)
  • 30. Survival Rates Related (n=65) and Unrelated (n=33) HSCT in Thalassemias OS Related vs Unrelated = 94% EFS Related = 88% EFS Unrelated = 82%
  • 31. MAC (n = 76) vs Novel RTC (n= 22) OS MAC = 95% RTC = 90% EFS MAC = 88% RTC = 93%
  • 32. MAC vs Novel RTC MAC RTC Acute GVHD gr III-IV 3 (4%) 2 (10%) Chronic GVHD Limited 6 (8%) 3 (13%) Extensive 2 (3%) 0 Dead 3 2 1 Graft failure 1 Fungal infection 1 Viral infection 1 Accident 1 Secondary AML
  • 33. Haploidentical HSCT (34+ selection) 22 pts Myeloablative regimen; Cyclo, Bu, Flu,Thiotepa and ATG GVHD prophylaxis CSA Sodani et al, Blood 2010
  • 34. All Thalassemia Patients May Be Cured with HSCT? TFS in related or unrelated for all age group (including pts age older than 10 yrs) in our center is 90%. Haploindentical HSCT in 12 severe patients (9/12 age > 10 yr) RTC plus Cyclophosphamide post transplant All 12 pts survived without thalassemia. No major complication except GvHD gr I-II. Follow up time; 4-20 months (abstract submitted to ASH 2014)
  • 36.
  • 37. Overview of the clinical protocol Testing and Release While Frozen Maximize % Transduced HSCs Vector + Cytokines Bone Marrow Harvest IV Infusion Transduced Cells (> 4x106 CD34+/Kg) (Spontaneous Homing) CD34+ cells (2x108 unsorted BM cells/Kg kept for rescue)
  • 38. Conclusion Outcomes of HSCT for thal in both MRD and MUD are favorable. Outcomes of HSCT for all age group among thal pts are also favorable. Whether the novel RTC regimen should be studied in younger thalassemia patients is needed to be investigated. Autologous HSC with gene addition may give a new hope for cure for thal patients.
  • 39. Acknowledgement Samart Pakakasama Ampaiwan Chuansumrit Nongnuch Sirachainan Usanarat Anurathapan Duantida Songdej HLA lab and BMT nurses Somtawain Sirirueng Wanpen Pantangkool Saengsuree Jootar Artit Ungkanont Vinai Suvattee Suthat Fucharoen Surapol Issaragrisil Borje Andersson Pramongkutklao Hospital Srinakarind Hospital Suandok Hospital Songklanakarin Hospital Siriraj Hospital
  • 40. Acknowledgement Ramathibodi Foundation Children Cancer Fund under The Patronage of HRH Princess Som Sawali

Hinweis der Redaktion

  1. HF information helps the registry to calculate the probability of finding a matched donor for patients. Many studies have shown that continued recruitment of random volunteers will increase the matched probability. Moreover, calculation of the percentage of patients for whom the donor was found was performed to evaluate unrelated donor search outcome. This figure show the percentage of patients who found matched donor when registry was grown up each year. Comparing the percentage of patients for whom the donor was found in the TSCDR with previous calculation other registries containing a pool of 10,000–23,000 donors, a potential HLA-A, -B, -DR serological split antigen matched donor was found in the TSCDR (49%), higher than the Korean Marrow Donor Program (28%) and the Europdonor Foundation (20%).
  2. We proposed sequential pretransplant immunosuppression, consists of fludarabine 40mg/squaremeter and dexamethasone 25 mg/squaremeter for 5 days, 28-day cylcle prior to conditioning regimen.
  3. This graph showdecreased CD4 cell prolifereation in all three representative patients.
  4. In the past, conditioning regimen for patients older than 10 y/o was a combination of cyclophosphamide and busulfan, which is too much alkylating agent and too toxic, therefor novel approach need to be explored
  5. Conditioning regimen comprised of 6-day of fludarabine, 4-day of busulfex and 3-day of antithymocyte globulin.
  6. Total eighteen patients were included in this study, two of them were second transplant. Four patients with homozygous beta thalassemia with median age at 14 y/o, seven of them were male and seven patients were undergone splenectomy. All patients had hepatomegaly, with high serum ferritin level.
  7. Most of them received peripheral blood stem cells, at median CD34 positive 9.4 million cell per kilogram. 11 of them were matched related donor.