Does Mixed Chimerism After Allogeneic Hematopoietic Cell Transplantation in Pediatric Patients With Fanconi Anemia Impact on Outcome?

Ayas, Mouhab; Siddiqui, Khawar; Al‐Jefri, Abdullah; Al-Ahmari, Ali; Ghemlas, Ibrahim; Alsaedi, Hawazen Saleh; Alanazi, Awatif; Khan, Saadiya; El-Solh, H; Al-Seraihi, Amal

doi:10.1016/j.jtct.2020.11.024

Cited by 8 publications

(8 citation statements)

References 35 publications

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“…All the other eleven patients remained with stable mixed chimerism, without any complications. Our results support a recent report showing mixed chimerism in patients with FA to have no adverse effect on the outcomes post‐HSCT 48 . Moreover, all patients who developed GVHD in our cohort had full‐donor chimerism, and none of the patients with mixed chimerism had any signs of GVHD at follow‐up.…”

Section: Discussionsupporting

confidence: 90%

“…Our results support a recent report showing mixed chimerism in patients with FA to have no adverse effect on the outcomes post‐HSCT. 48 Moreover, all patients who developed GVHD in our cohort had full‐donor chimerism, and none of the patients with mixed chimerism had any signs of GVHD at follow‐up. This correlation was shown before among patients with aplastic anemia, including a small cohort of patients with FA.…”

Section: Discussionmentioning

confidence: 72%

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Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes

Fink

Even‐Or

Avni

et al. 2022

Clinical Transplantation

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Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for the hematological complications of patients with Fanconi anemia (FA). Over the last two decades, HSCT outcomes have improved dramatically following the development of regimens tailored for FA patients. In this study, we analyzed genetic, clinical, and transplant data of 41 patients with FA who underwent HSCT at Hadassah Medical Center between November 1996 and September 2020. Overall survival (OS) was 82.9% with a median follow-up time of 2.11-years (95% CI, .48-16.56).Thirteen patients (31.7%) developed acute graft-versus-host disease (GVHD), three of them with grades 3-4. Nine patients developed chronic GVHD, five had extensive disease. Twelve patients (29.3%) developed stable mixed-chimerism with complete resolution of bone marrow failure (BMF); none of them had acute nor chronic GVHD. Significantly higher GVHD rates were observed in transplants from peripheral blood stem cell grafts as compared to other stem cell sources (p = .002 for acute and p = .004 for chronic GVHD). Outcome parameters were comparable between HSCT from matched-sibling (n = 20) to other donors (n = 21), including survival rates (p = .1), time to engraftment (p = .69 and p = .14 for neutrophil and platelet engraftment time, respectively), chimerism status (p = .36 and p = .83 for full-donor and mixed chimerism, respectively), and GVHD prevalence (p = 1). Our results demonstrate the vast improvements in HSCT outcomes of patients with FA, narrowing the gap between matched-sibling versus alternative donor transplantations. Our data identifies factors that may significantly affect transplant outcomes such as graft source and chimerism status.

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Section: Discussionsupporting

confidence: 90%

Section: Discussionmentioning

confidence: 72%

Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes

Fink

Even‐Or

Avni

et al. 2022

Clinical Transplantation

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“…In ALD, ≥ 70% myeloid cell chimerism in the early period after HCT is necessary for rapid improvement of radiological findings [57]. Stable MC did not affect clinical outcomes in patients with FA, although its contribution to clonal evolution from the remnant recipient cells remains unclear [58]. Myeloid cell chimerism < 30% following allogeneic HCT for sickle cell disease was found sufficient to prevent vaso-occlusive crises in hematologically normal donors, whereas myeloid cell chimerism ≥ 50% was required to correct hemolysis [59].…”

Section: Therapeutic Intervention For MCmentioning

confidence: 99%

Unresolved issues in allogeneic hematopoietic cell transplantation for non-malignant diseases

Umeda

2022

Int J Hematol

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Allogeneic hematopoietic cell transplantation (HCT) can be curative for a variety of non-malignant diseases (NMDs) as well as hematological malignancies. However, there are several fundamental differences between HCT for NMDs and hematological malignancies, which may necessitate the use of alternative HCT strategies. For example, these diseases differ in the intensity of conditioning regimen sufficient to improve disease. In addition, patients with NMDs are at higher risk of graft failure or mixed chimerism following HCT, and gain no or little survival benefit from graft-versus-host disease. Because more than 80% of patients with NMDs become long-term survivors, greater attention has been paid to late adverse effects and decreased of quality of life after HCT. This review addresses several unresolved issues in allogeneic HCT for patients with NMDs, such as (1) stem cell source, (2) conditioning regimen, (3) use of serotherapy or low-dose irradiation, and (4) therapeutic intervention for mixed chimerism. Resolving these issues may improve transplant outcomes in patients with NMDs.

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“…MC in the T-cell lineage is frequent and has no consequences for disease cure, whereas high-level myeloid engraftment is essential. 73,74…”

Section: Bone Marrow Failurementioning

confidence: 99%

“…These patients often receive minimal intensity conditioning due to the hypoplastic state of their bone marrows. MC in the T‐cell lineage is frequent and has no consequences for disease cure, whereas high‐level myeloid engraftment is essential 73,74 …”

Section: The Significance Of Cell Lineage Chimaerism In Paediatric Ha...mentioning

confidence: 99%

The significance of mixed chimaerism and cell lineage chimaerism monitoring in paediatric patients post haematopoietic stem cell transplant

2022

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Haematopoietic stem cell transplants (HSCTs) are carried out across the world to treat haematological and immunological diseases which would otherwise prove fatal. Certain diseases are predominantly encountered in paediatric patients, such severe primary immunodeficiencies (PID) and diseases of inborn errors of metabolism (IEM). Chimaerism testing for these disorders has different considerations compared to adult diseases. This review focuses on the importance of cell-lineagespecific chimaerism testing and examines the appropriate cell populations to be assessed in individual paediatric patient groups. By analysing disease-associated subpopulations, abnormalities are identified significantly earlier than in whole samples and targeted clinical decisions can be made. Chimaerism methods have evolved over time and lead to an ever-increasing level of sensitivity and biomarker arrays to distinguish between recipient and donor cells. Short tandem repeat (STR) is still the gold standard for routine chimaerism assessment, and hypersensitive methods such as quantitative and digital polymerase chain reaction (PCR) are leading the forefront of microchimaerism testing. The rise of molecular methods operating with minute DNA amounts has been hugely beneficial to chimaerism testing of paediatric samples. As HSCTs are becoming increasingly personalised and risk-adjusted towards a child's individual needs, chimaerism testing needs to adapt alongside these medical advances ensuring the best possible care.How to cite this article: Kricke S, Rao K, Adams S. The significance of mixed chimaerism and cell lineage chimaerism monitoring in paediatric patients post haematopoietic stem cell transplant.

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Does Mixed Chimerism After Allogeneic Hematopoietic Cell Transplantation in Pediatric Patients With Fanconi Anemia Impact on Outcome?

Cited by 8 publications

References 35 publications

Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes

Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes

Unresolved issues in allogeneic hematopoietic cell transplantation for non-malignant diseases

The significance of mixed chimaerism and cell lineage chimaerism monitoring in paediatric patients post haematopoietic stem cell transplant

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