Stem cell transplantation in India

Chandy, Mammen

doi:10.1038/bmt.2008.124

Cited by 54 publications

(36 citation statements)

References 8 publications

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“…This is the first report on a large series of thalassemia patients undergoing BMT exclusively in startup centers and directly in LMICs and might provide a relevant precedent to address the dire shortage of such services in emerging countries, 52 where hemoglobinopathies are the most frequent life-threatening noncommunicable disease among children 1 and a major indication for BMT. 6,7,53 Adding Tt 54 or ATG 13,17,18,19 to the BuCy backbone has substantially reduced rejection rates. In the age group ,4 years of age, where Tt seems particularly important to reduce rejection, 15 we observed no significant rejection differences between the ATG and Tt groups.…”

Section: Discussionmentioning

confidence: 99%

“…ST is also a major indication for blood and marrow transplantation (BMT) in children in the Indian subcontinent. [6][7][8] BMT is currently the only definitive curative option with success rates .80% 9 and potential normalization of health-related quality of life (HRQoL) 10 in low-risk children with a compatible related donor. The cost of BMT is equivalent to that of a few years of noncurative supportive care.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

ATG vs thiotepa with busulfan and cyclophosphamide in matched-related bone marrow transplantation for thalassemia

et al. 2017

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Key Points• In low-risk BMT for thalassemia, ATGBuCy seems as effective as Tt-BuCy in the prevention of rejection and may decrease transplant-related mortality. received ATG-BuCy. All patients were ,15 years and had no hepatomegaly (liver #2 cm from costal margin). Actuarial overall survival in the Tt-BuCy and ATG-BuCy groups was 87% and 94% and thalassemia-free survival was 80% and 85% at a median follow-up of 37 and 17 months, respectively, with no significant differences by log-rank statistics. SubstitutingTt with ATG in the standard BuCy context seems safe and effective and may decrease transplant-related mortality. Higher fertility rates are expected for patients who received ATG-BuCy.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

ATG vs thiotepa with busulfan and cyclophosphamide in matched-related bone marrow transplantation for thalassemia

et al. 2017

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“…10 In areas were thalassemia is endemic there is a severe shortage of centers for its cure. 11,12 This is not only due to lack of financial resources, in fact BMT is less expensive compared to long-term supportive care, 13 but other factors may contribute such as lack of specific professional expertise or the perception that setting up a BMT center is highly complex and expensive.…”

Section: Thalassemia Disease Burdenmentioning

confidence: 99%

Transplantation in Low Resource Countries

Faulkner¹,

Yaqub

Khalid³

et al. 2011

Thalassemia Reports

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Thalassemia major (TM) is the most common deadly genetic disorder, a major cause of chronic non-infectious morbidity and financial burden in many low and middle-income regions. In these settings few children reach adulthood because proper long-term supportive care is seldom available. Bone marrow transplantation (BMT) is the only available curative modality and it can be very successful and cost-effective for young children with low-risk features and a compatible related donor. However, in countries where TM is most prevalent, there is a dire shortage of BMT centers. The Cure2Children Foundation has supported a feasibility study evaluating safety, efficacy and costs of developing a new BMT center in an underserved lower-middle-income country with relatively untrained professionals within a structured collaboration and knowledge-transfer program. A total of 24 consecutive patients who underwent BMT in Pakistan between September 2008 and August 2010 are included in this prospective analysis, 17 from an established bone marrow transplant center, the National Institute for Blood Diseases in Karachi, Pakistan and the initial 7 BMTs from a start up unit in a government civil hospital, the Pakistan Institute of Medical Sciences Children's Hospital in Islamabad. Patients were matched for age, nutritional status, growth, disease, disease status and post-BMT follow-up time. All patients had a matched-related sibling donor, were younger than 10 years of age at the time of transplantation, received the same conditioning regimen. All needy families could rely on a support program throughout the 8-month post-transplant period. The Cure2Children Foundation provided professional and financial support as well as a structured web-based data management and cooperation platform. At a median follow up of 19.6 months (range 8.7 to 31.5) actuarial thalassemia-free survival is 85.6% and 85.7% and overall survival 94.1% and 85.7% in the established and start-up center respectively with no statistically significant differences. Other outcome indices like infectious complications, engraftment parameters, transplant-related complications, and post-BMT performance scores also did not differ. The median cost of matched-related transplants in the start-up center, including pre-BMT evaluation, was 11,513 USD (range 7,518 to 21,176). Within structured cooperation strategies bone marrow transplantation for thalassemia major can be performed safely, effectively, and affordably even in start-up centers in lower-middleincome countries, like Pakistan, were most thalassemia patients live. This observation may have important implications to increase access to cure for thalassemia worldwide.

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“…Recent reports on HSCT from Asian countries suggest the progress of HSCT in this region. [5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20][21] However, no comprehensive picture of the current state of HSCT in Asia has been obtained. The Asia-Pacific region consists of countries with various ethnic, economical and social backgrounds.…”

Section: Introductionmentioning

confidence: 99%