Allogeneic Bone Marrow Transplant as a Cure for Refractory T-Cell Large Granular Lymphocytic Leukemia in an Adolescent

Fries, Carol; Evans, Andrew; Cheon, HeeJin; Korones, David N.; Loughran, Thomas P.; Andolina, Jeffrey R.

doi:10.1097/mph.0000000000002390

Cited by 2 publications

(6 citation statements)

References 24 publications

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“…Nine patients with refractory LGL leukemia and rheumatoid arthritis were treated with tofacitinib and most had marked improvement in neutrophil count 30 . HSCT has been reported in two patients with refractory T‐LGL resulting in a cure, suggesting that HSCT may be a potential therapy for patients with difficult to treat disease 47,113 …”

Section: Stat3‐related Diseasementioning

confidence: 99%

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JAK/STAT defects and immune dysregulation, and guiding therapeutic choices

Chaimowitz,

Smith,

Forbes Satter

2024

Immunological Reviews

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SummaryInborn errors of immunity (IEIs) encompass a diverse spectrum of genetic disorders that disrupt the intricate mechanisms of the immune system, leading to a variety of clinical manifestations. Traditionally associated with an increased susceptibility to recurrent infections, IEIs have unveiled a broader clinical landscape, encompassing immune dysregulation disorders characterized by autoimmunity, severe allergy, lymphoproliferation, and even malignancy. This review delves into the intricate interplay between IEIs and the JAK–STAT signaling pathway, a critical regulator of immune homeostasis. Mutations within this pathway can lead to a wide array of clinical presentations, even within the same gene. This heterogeneity poses a significant challenge, necessitating individually tailored therapeutic approaches to effectively manage the diverse manifestations of these disorders. Additionally, JAK–STAT pathway defects can lead to simultaneous susceptibility to both infection and immune dysregulation. JAK inhibitors, with their ability to suppress JAK–STAT signaling, have emerged as powerful tools in controlling immune dysregulation. However, questions remain regarding the optimal selection and dosing regimens for each specific condition. Hematopoietic stem cell transplantation (HSCT) holds promise as a curative therapy for many JAK–STAT pathway disorders, but this procedure carries significant risks. The use of JAK inhibitors as a bridge to HSCT has been proposed as a potential strategy to mitigate these risks.

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Section: Stat3‐related Diseasementioning

confidence: 99%

“…30 HSCT has been reported in two patients with refractory T-LGL resulting in a cure, suggesting that HSCT may be a potential therapy for patients with difficult to treat disease. 47,113…”

Section: Somatic Stat3 Gain Of Functionmentioning

confidence: 99%

JAK/STAT defects and immune dysregulation, and guiding therapeutic choices

Chaimowitz,

Smith,

Forbes Satter

2024

Immunological Reviews

View full text Add to dashboard Cite

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“…Treatment is indicated for patients with symptomatic anemia, neutropenia with recurrent infections, and/or autoimmune conditions [ 203 ]. Immunosuppressive therapy (commonly methotrexate, cyclosporine, and cyclophosphamide) often mitigates sequelae but may or may not result in cure [ 194 , 203 ]. In addition to immunosuppressive therapy, chemotherapy or HSCT may also be utilized [ 194 , 200 , 204 , 205 , 206 ].…”

Section: T-cell Lymphoid Proliferations Rare Entitiesmentioning

confidence: 99%

“…Immunosuppressive therapy (commonly methotrexate, cyclosporine, and cyclophosphamide) often mitigates sequelae but may or may not result in cure [ 194 , 203 ]. In addition to immunosuppressive therapy, chemotherapy or HSCT may also be utilized [ 194 , 200 , 204 , 205 , 206 ]. In the relapsed/refractory setting, allo-HSCT successfully induced remission in a 13-year-old, suggesting allo-HSCT be considered for a definitive cure in children [ 194 ].…”

Section: T-cell Lymphoid Proliferations Rare Entitiesmentioning

confidence: 99%

“…In addition to immunosuppressive therapy, chemotherapy or HSCT may also be utilized [ 194 , 200 , 204 , 205 , 206 ]. In the relapsed/refractory setting, allo-HSCT successfully induced remission in a 13-year-old, suggesting allo-HSCT be considered for a definitive cure in children [ 194 ]. Molecular pathways implicated in T-LGL pathogenesis offer potential for targeted therapeutic approaches [ 203 ], though none have been studied in children and AYAs.…”

Section: T-cell Lymphoid Proliferations Rare Entitiesmentioning

confidence: 99%

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Rare Hematologic Malignancies and Pre-Leukemic Entities in Children and Adolescents Young Adults

Brown,

Batra

2024

Cancers

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There are a variety of rare hematologic malignancies and germline predispositions syndromes that occur in children and adolescent young adults (AYAs). These entities are important to recognize, as an accurate diagnosis is essential for risk assessment, prognostication, and treatment. This descriptive review summarizes rare hematologic malignancies, myelodysplastic neoplasms, and germline predispositions syndromes that occur in children and AYAs. We discuss the unique biology, characteristic genomic aberrations, rare presentations, diagnostic challenges, novel treatments, and outcomes associated with these rare entities.

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Allogeneic Bone Marrow Transplant as a Cure for Refractory T-Cell Large Granular Lymphocytic Leukemia in an Adolescent

Cited by 2 publications

References 24 publications

JAK/STAT defects and immune dysregulation, and guiding therapeutic choices

JAK/STAT defects and immune dysregulation, and guiding therapeutic choices

Rare Hematologic Malignancies and Pre-Leukemic Entities in Children and Adolescents Young Adults

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