Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders

Marsh, Rebecca; Hebert, Kyle; Kim, Soyoung; Dvorak, Christopher C.; Aquino, Victor; Baker, K. Scott; Chellapandian, Deepak; Saldaña, Blachy Dávila; Duncan, Christine; Eckrich, Michael J.; Georges, George E.; Olson, Timothy S.; Pulsipher, Michael A.; Shenoy, Shalini; Stenger, Elizabeth; Lugt, Mark Vander; Yu, Lolie C.; Gennery, Andrew R.; Eapen, Mary

doi:10.1016/j.jaci.2021.07.031

Cited by 23 publications

(11 citation statements)

References 28 publications

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“…1 Chemotherapy protocols for major RIC regimen listed in the EBMT/ESID inborn errors working party guidelines [2]. HCT hematopoietic cell transplantation, BU busulfan, FLU fludarabine, TREO treosulfan, TEPA thiotepa, MEL melphalan, CY cyclophosphamide combined with FLU as a less toxic RIC [2,3,39]. Furthermore, gonadal dysfunction seems to be less severe in females undergoing HCT with FLU+MEL [40].…”

Section: Conditioning Regimenmentioning

confidence: 99%

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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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Section: Conditioning Regimenmentioning

confidence: 99%

“…Furthermore, gonadal dysfunction seems to be less severe in females undergoing HCT with FLU+MEL [40]. Drawbacks to the use of FLU+MEL include higher rates of MC, viral reactivation, and cardiac toxicities was a major concern [38,39,41].…”

Section: Conditioning Regimenmentioning

confidence: 99%

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

Umeda

2022

Int J Hematol

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“…after HSCT [44]. Earlier studies using myeloablative conditioning showed poor outcome after HSCT because of pre-existing multiorgan toxicity and infections before HSCT, and incidence of hepatic venoocclusive disease.…”

Section: Wiskott-aldrich Syndrome (Was)mentioning

confidence: 99%

“…The use of RIC containing fludarabine, melphalan, and alemtuzumab or thiotepa, and better HLH control prior to conditioning were associated with better outcome [29,44]. The use of RIC is further supported by the fact that a donor chimerism ＞20-30% was protective against late reactivation.…”

Section: Wiskott-aldrich Syndrome (Was)mentioning

confidence: 99%

How I Treat Primary Immune Deficiencies with Hematopoietic Stem Cell Transplantation

Kook

Kim

Baek

2022

Clin Pediatr Hematol Oncol

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Primary immune deficiencies (PID), or more recently, inborn errors of immunity (IEI), resulting from genetic defects of the immune system may present with increased susceptibility to infections, persistent inflammation, and autoimmunity. With recent introduction of next generation sequencing, the number of IEIs increases rapidly, reaching to 484 in 2022. Hematopoietic stem cell transplantation (HSCT) has been used over decades as a mainstay of specific treatment modality, while gene therapy and pharmacologic approach have been attempted with promising results in some PID in recent years. The survival following allogeneic HSCT for PID is now generally ＞80%. The indication and timing of transplant must be individualized not only on the basis of the specific PID but also on the characteristics of the individual patient. For the successful transplant outcome, the choice of donor and the optimal pretransplant conditioning regimen is important. This article will discuss current status and recommendations from specialists in HSCT for some representative PID, including severe combined immunodeficiency (SCID), CD40 ligand or CD40 deficiency, Wiskott-Aldrich syndrome, hemophagocytic lymphohistiocytosis, and chronic granulomatous disease, along with our personal experience of PID treatment in Korea.

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“…Striking an appropriate balance between intensity of conditioning regimen for HSCT and disease control remains a challenge. 8,9 Consequently, only through careful study of strategies among similar disease groups and/or donor types (in the case for transplantation) can we begin to make appropriate recommendations for treatments.…”

mentioning

confidence: 99%

To treat with curative intent or modify disease?

Eapen

2023

Blood

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Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders

Cited by 23 publications

References 28 publications

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

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

How I Treat Primary Immune Deficiencies with Hematopoietic Stem Cell Transplantation

To treat with curative intent or modify disease?

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