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Towards new long-term composite ‘Quality of Survival’ endpoints

Bone Marrow Transplantation volume 55, pages 1898–1899 (2020)Cite this article

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Saglio et al. describe in this issue the late effects of hematopoietic cell transplantation (HCT) in children affected by acute leukemia receiving either a Busulfan-based or TBI-based conditioning regimen. They are presenting data from the ‘Associazione Italiana Ematologia Oncologia Pediatrica’ (Italian Pediatric Hematology and Oncology Group) [1]. This is a timely and a super important contribution to the literature. Where decades ago, survival after HCT was already a great outcome, nowadays this is not enough. The ‘quality of survival’ (over time), impacted by the occurrence of late effects, late mortality, is probably a better long-term composite endpoint. Some of the events, potentially significantly impacting the ‘quality of life’ and even survival, can occur decades after treatment. Longer follow-up adds more burden, up to 60% 10 years after HCT [2, 3]. Therefore, also, the participation of these HCT survivors in the society and day-to-day life (e.g., school and employment), on the long term, should be considered. This is first of all important to counsel the families before HCT, but obviously also for providers and researchers to identify remaining needs to further optimize treatment solutions.

In the article by Saglio et al. in this issue, not surprisingly, TBI was found to a predictor for late effects, such as cataract and secondary malignancies. Where cataract may not be considered a severe late effect (relatively easy fix; but a burden), a second malignancy is a significant life-threatening burden. At ~15 years (longest follow-up), the difference in occurrence of second malignancies is about 20% higher in the TBI-based group compared to Bu-based conditioning. And the curve has not plateaued yet. If we would consider a secondary malignancy as an event in a composite long-term outcome (@10 or 20 years for instance) only this event will already have a significant effect on long-term ‘quality of survival’ curve. Contrary to some other late effect studies, in this study no impact on growth and gonadal function abnormalities were found. However, the median follow-up was 7 years only. This may have contributed. An endpoint that has not been measured over time in these retrospective analyses, is the evolution of IQ. Others have clearly shown that patients receiving TBI show a gradual, but not insignificant decline in IQ over time [4, 5]. This endpoint may also be of importance for participation and contribution in the society and may significantly impact the ‘quality of life.’ All the burdens described above, can potentially impact the ‘quality of life,’ therefore, adding over time measuring the ‘quality of life’ would very helpful to get the full picture of the outcomes.

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References

  1. Saglio F, Zecca M, Pagliara D, Giorgiani G, Balduzzi A, Calore E, et al. Occurrence of long-term effect after hematopoietic stem cell transplantation in children affected by acute leukemia receiving either busulfan or total body irradiation: results of an AIEOP retrospective study. Bone Marrow Transplant. 2020. https://doi.org/10.1038/s41409-020-0806-8. Online ahead of print.

  2. Eissa HM, Lu L, Baassiri M, Bhakta N, Ehrhardt MJ, Triplett BM, et al. Chronic disease burden and frailty in survivors of childhood HSCT: a report from the St. Jude Lifetime Cohort Study. Blood Adv. 2017;1:2243–6.

    Article  Google Scholar 

  3. Lawitschka A, Peters C. Long-term effects of myeloablative allogeneic hematopoietic stem cell transplantation in pediatric patients with acute lymphoblastic leukemia. Curr Oncol Rep. 2018;20:74–10.

    Article  Google Scholar 

  4. Krull KR, Zhang N, Santucci A, Srivastava DK, Krasin MJ, Kun LE, et al. Long-term decline in intelligence among adult survivors of childhood acute lymphoblastic leukemia treated with cranial radiation. Blood. 2013. https://doi.org/10.1182/blood-2013-03-487744.

  5. Willard VW, Leung W, Huang Q, Zhang H, Phipps S. Cognitive outcome after pediatric stem-cell transplantation: impact of age and total-body irradiation. J Clin Oncol. 2014;32:3982–8.

    Article  Google Scholar 

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  1. Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Jaap Jan Boelens

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  1. Jaap Jan Boelens
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Correspondence to Jaap Jan Boelens.

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I have no conflict of interest related to this topic. Unrelated I am a consulted for Takeda, Magenta, Omeros, Advanced Clinical, Bluebird Bio, and Bluerock

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Boelens, J.J. Towards new long-term composite ‘Quality of Survival’ endpoints. Bone Marrow Transplant 55, 1898–1899 (2020). https://doi.org/10.1038/s41409-020-0883-8

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