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Haploidentical Stem Cell Transplant with Post-Transplant Cyclophosphamide in Pediatric Hemophagocytic Lymphohistiocytosis

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Abstract

Purpose

Primary hemophagocytic lymphohistiocytosis is a severe and uncommon disease affecting pediatric patients. Genetic abnormalities have been related to altered apoptosis and exaggerated inflammatory reactions. Chemoimmunotherapy and stem cell transplantation are treatment options, but transplant is the only curative treatment. Here we aim to describe the treatment with hematopoietic stem cell transplantation with a novel strategy and the outcomes.

Methods

An observational, descriptive, case series study was performed in pediatric patients of two high complexity medical centers in Colombia. Data was collected retrospectively between 2015 and 2020.

Results

We describe five pediatric cases with a diagnosis of primary hemophagocytic lymphohistiocytosis. All were treated with replete-cell haploidentical hematopoietic stem transplantation, reduced-intensity conditioning, and post-transplant cyclophosphamide, in two high-complexity centers in Colombia. All patients are alive, and one is receiving management for chronic graft-versus-host disease.

Conclusion

To the best of our knowledge, there are few reports in the literature with this strategy, promising a possible alternative when there are no other donor options.

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Notes

  1. Fundación Valle de Lili (FVL) in Cali and Hospital Pablo Tobón Uribe (HPTU) in Medellín

Abbreviations

HLH:

hemophagocytic lymphohistiocytosis

FHLH:

familial hemophagocytic lymphohistiocytosis

HSCT:

hematopoietic stem cell transplantation

MSD:

matched sibling

MUD:

matched unrelated donor

PTCy:

post-transplantation cyclophosphamide

FVL:

Fundación Valle de Lili

HPTU:

Hospital Pablo Tobón Uribe

EBV:

Ebstein-Barr virus

CMV:

cytomegalovirus

GvHD:

graft-vs-host disease

aGVHD:

acute graft-vs-host disease

cGVHD:

chronic graft-vs-host disease

GI:

gastrointestinal

VOD:

veno-occlusive disease

TA-TMA:

transplant-associated thrombotic microangiopathy

TRM:

transplant-related mortality

References

  1. Marsh RA, Haddad E. How I treat primary haemophagocytic lymphohistiocytosis. Br J Haematol. 2018;182(2):185–99.

    Article  Google Scholar 

  2. Marsh RA, Vaughn G, Kim M-O, Li D, Jodele S, Joshi S, et al. Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood. 2010;116(26):5824–31 Available from: https://ashpublications.org/blood/article/116/26/5824/28172/Reducedintensity-conditioning-significantly.

  3. Ouachée-Chardin M, Elie C, De Saint BG, Le Deist F, Mahlaoui N, Picard C, et al. Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients. Pediatrics. 2006;117(4):e743–50.

    Article  Google Scholar 

  4. Jia C, Wang B, Zhu G, Zhang R, Wang K, Yan Y, et al. Haploidentical hematopoietic stem cell transplantation using reduced-intensity conditioning for pediatric patients with familial hemophagocytic lymphohistiocytosis. Pediatr Investig. 2018;2(4):216–21. https://doi.org/10.1002/ped4.12096.

    Article  PubMed  Google Scholar 

  5. Uppuluri R, Sivasankaran M, Patel S, Swaminathan VV, Ramanan KM, Ravichandran N, et al. Haploidentical stem cell transplantation with post-transplant cyclophosphamide for primary immune deficiency disorders in children: challenges and outcome from a tertiary care center in South India. J Clin Immunol. 201918;39(2):182–7 Available from: http://ovidsp.ovid.com/ovidweb.cgi?T=JS&PAGE=reference&D=prem&NEWS=N&AN=30778805.

  6. Kohli S, Rastogi N, Nivargi S, Thakkar D, Katewa S, Yadav SP. Successful haploidentical stem cell transplant with posttransplant cyclophosphamide for hemophagocytic lymphohistiocytosis. J Pediatr Hematol Oncol. 2019;41(3):E158–60.

    Article  CAS  Google Scholar 

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Author information

Authors and Affiliations

  1. Maternal and Child Department, Pediatric Stem Cell Transplant Service, Fundación Valle del Lili, Carrera 98 #18-49, Cali, 760032, Colombia

    Diego Medina-Valencia & Alexis A. Franco

  2. School of Medicine, Universidad Icesi, Cali, Colombia

    Diego Medina-Valencia, Alexis A. Franco & Manuela Olaya

  3. Maternal and Child Department, Fundación Valle del Lili, Cra 98 No. 18 – 49, Cali, 760032, Colombia

    Daniela Cleves

  4. Centro de Investigaciones Clínicas, Fundación Valle del Lili, Cra 98 No. 18 – 49, Cali, 760032, Colombia

    Daniela Cleves & Estefania Beltran

  5. Pediatric Stem Cell Transplant Unit, Hospital Pablo Tobón Uribe, Cll 78b #69-240, Medellín, 11001, Colombia

    Natalia Builes & Andrés Felipe Escobar-González

  6. Maternal and Child Department, Allergology and Immunology Unit, Fundación Valle del Lili, Cra 98 No. 18 – 49, Cali, 760032, Colombia

    Manuela Olaya

Authors
  1. Diego Medina-Valencia
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  2. Daniela Cleves
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  3. Estefania Beltran
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  4. Natalia Builes
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  5. Alexis A. Franco
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  6. Andrés Felipe Escobar-González
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  7. Manuela Olaya
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Contributions

All authors contributed to the study conception and design. Material preparation and data collection were performed by DC, NB, and EB. Critical revision and analysis were performed by MO, NB, AAF, AFE, and DMV. The first draft of the manuscript was performed by DC and EB. All authors commented on previous versions of the manuscript and read and approved the final manuscript version to be published. Also, agreement to be accountable for all aspects of the work and to ensure that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Corresponding author

Correspondence to Diego Medina-Valencia.

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Conflict of Interest

The authors declare no conflict of interest.

Ethics Approval

The study was approved by the Institutional Ethics Committee in accordance with the Declaration of Helsinki.

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Medina-Valencia, D., Cleves, D., Beltran, E. et al. Haploidentical Stem Cell Transplant with Post-Transplant Cyclophosphamide in Pediatric Hemophagocytic Lymphohistiocytosis. J Clin Immunol 41, 1172–1177 (2021). https://doi.org/10.1007/s10875-021-01009-3

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  • DOI: https://doi.org/10.1007/s10875-021-01009-3

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