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Bone Marrow Transplantation 3rd Pan-European Conference on Haemoglobinopathies & Rare Anaemias Limassol, 24 – 26 October 2012 Lawrence Faulkner Cure2Children.

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Presentation on theme: "Bone Marrow Transplantation 3rd Pan-European Conference on Haemoglobinopathies & Rare Anaemias Limassol, 24 – 26 October 2012 Lawrence Faulkner Cure2Children."— Presentation transcript:

1 Bone Marrow Transplantation 3rd Pan-European Conference on Haemoglobinopathies & Rare Anaemias Limassol, 24 – 26 October 2012 Lawrence Faulkner Cure2Children Foundation-Italy

2 Bone marrow and stem cell transplantation BMT in the “new thalassemic” era  Mortality and morbidity  Quality of life  Cost-effectiveness  Global perspective

3 Risk Classes for BMT in Thalassemia Standard preparation with Busulfan-Cyclophosphamide (BuCy) ChelationHepatomegalyFibrosis Class 1RegularNo Class 2Reg/IrregNo/yesNo/Yes Class 3IrregularYesyes OSTFS Class 194%87% Class 284%81% Class 370%58% Patients < 17 year Adults OSTFS 67%63% Emanuele Angelucci: Hematopoietic stem cell transplantation for Thalassemia. ASH Educational Book 2010

4 Bone marrow and stem cell transplantation Late effects  Infertility  Delayed puberty (females) Impaired growth and development (TBI) Second malignancies (TBI) Intellectual impairment (CNS-directed therapy)

5 Caocci G, et al. EBMT 2012: P1034 54 patients (26 females and 28 males) after a mean of 20 years from sibling donor BMT compared to 41 sibling donors. Mean age at BMT 14 yrs (currently 38 yrs) and 18 (currently 41 yrs) for patients and donors, respectively. Chronic graft versus host disease reported in 18.5%. Patients had lower QoL scores for vitality, physical & social functioning PatientDonor sibling Working67%80% Living with spouse/partner40%61% Had babies15%31% Quality of life post-BMT

6 Splenectomy and BMT  Enlarged spleen per se is not associated with higher rejection rates  May increase transfusion requirements and delay engraftment  Splenectomy may be associated with increased transplant- related mortality. Mathews et al. Pediatr Transplant. 2009;13(2):171–176 Bone marrow and stem cell transplantation

7 Hepatitis C and BMT  No evidence that BMT has any influence on the course of HCV infection or vice versa.  Hepatitis C and iron overload may accelerate progression of liver fibrosis Erer et al. Bone Marrow Transplant.,1994;14(3):369–372 Angelucci et al. Blood, 2002;100(1):17–21 HCV positivity may thus actually strengthen the indication for BMT if proper supportive care is not available Bone marrow and stem cell transplantation

8 Cost-effectiveness A cost-utility and budget impact analysis of allogeneic hematopoietic stem cell transplantation for severe thalassemic patients in Thailand Leelahavarong et al. BMC Health Services Research 2010, 10:209 ( Bone marrow and stem cell transplantation

9 A global perspective

10 Thalassemia in Pakistan Thalassemia carriers5% population Total Carriers 8 million Thalassaemia births/year 5,000 – 6,000 Thalassaemia Major prevalence~ 60,000 ~ 4% national health budget goes to thalassemia care (for appropriate management it would have to increase to 40%). Thalassemia is the main donated blood consumer

11 Thalassemia Pakistan vs. UK UKPakistan Prevalence70060.000 New cases/year305.000

12 Age Groups 0-10 years : 75.6% 11-20 years: 24.1% 21-40 years: 0.3% Thalassaemia statistics in Pakistan

13 1996: data from HBB Karachi, 2006: data from BTIHS, Karachi Thalassemia survival in Pakistan


15 Mission Cure2Children (C2C) is a non-profit, apolitical, and secular organization providing support for the care of children with cancer and severe blood disorder in developing countries.

16 Ayesha 8 y/o with thalassemia transplanted in Italy Pakistan

17 BMT for thalassemia Irfan M et al., JPMA 2008 60% Infections? Skills? Patient selection? Family support? Lucarelli G at al., 500 class 1-2 pts 85%


19 Objectives of the Pakistan project 1.> 80% cure il low-risk patients with a matched sibling 2.Increase awareness and access to cure, screening and prevention

20 Prospective (clinical management-oriented) database

21 Computerized treatment plans (Excel format)








29 Local personnel

30 Jaipur Rajhastan-India





35 Bone marrow and stem cell transplantation A global perspective Outcome of the first 12 low risk patients age <6 y, liver <2cm, matched sibling donor:  6 at PIMS-Pakistan  6 at SEAIT-Jaipur 92% cure

36 Bone marrow and stem cell transplantation A global perspective Construction & Renovation Furniture & Equipments Total PIMS Pakistan11,000$24,000$35,000$ SEAIT India135,000$29,000$164,000$

37 Bone marrow and stem cell transplantation Costs (low-risk, compatible sibling) Pre-BMTevaluation Diagnostics Blood products Hospital charges Surgical Pharmacy Professionals Follow up Total Pakistan (PIMS) $400$1,700$200$600$300$2,100$4,600$900$10,700 India (SEAIT) $500$2,500$200$3,100$1,800$4,600$1,200$900$15,000




41 Indirect positive effects of BMT  Increases awareness about thalassemia  Attracts families for screening  Generates resources for supportive care and prevention  Increase compliance with chelation and use of safe blood  Attracts and motivate professionals  Empowers medical centers and paves the way for the cure of related disorders

42 Accessible HLA screening Poster session, El Missiry et al.: HLA-typing by buccal swab to facilitate acees to bone marrow transplantation globally. Abstract book page 104

43 Bone marrow and stem cell transplantation Thalassemia vs. Sickle cell disease ThalassemiaSickle cell disease New cases/year> 80,000> 250,000 Total BMTs done~ 3,000~ 300 Risk group identification GoodPoor Transplant related mortality 5-40%5-10% Medical therapy Chelation effective but expensive Hydroxyurea inexpensive but partially effective Quality of life Potentially goodVariable Life expectancy Highly dependent on medical care Potentially good with modest care

44 Prospects Partially matched related (haploidentical) BMT for sickle cell disease MortalityIII-IV GVHDGraft failure Haplo BMT (14 pts: 15-42 yrs) 0% 43% Bolanos-Meade et al. Blood 2012;118:282

45 Prospects Double unrelated cord blood vs. Haploidentical marrow Results of 2 parallel phase 2 trials (adult leukemia/lymphoma pts ) 1-yr non- relapse mortality d100 II-IV GVHD Median ANC recovery (>500) Median plt recovery (>20K) Graft failure Stem cell source cost dUCB (50 pts) 24%40%d15d3012% >60.000$ Haplo BMT (50 pts) 7%32%d16d242% 0 Brunstein et al. Blood 2011;118:282

46 The role of third-party international professional organizations  Foster the use of common protocols and procedures  Reassure patients and professionals in the early phase  Decrease unnecessary expenses  Increased scientific visibility  Support fund-development  Provide network referrals  Promote screening and prevention

47 Bone marrow and stem cell transplantation Conclusions  BMT outcomes are improving in parallel with supportive and thus indications in severe thalassemia are still controversial, at least in affluent countries  In thalassemia-prone regions where appropriate long-term supportive care might not be readily accessible and resources limited, BMT is feasible, cost-effective and can save lives.  Thalassemia associations, NGOs and professional networks may play an important role to promote non-profit and ethical BMT programs  In emerging countries better access to BMT may increases hope and thus improve compliance with supportive care  BMT programs may increase awareness, attract professionals and promote prevention

48 Thank you

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