sirolimus and Leukemia--Myelomonocytic--Juvenile

sirolimus has been researched along with Leukemia--Myelomonocytic--Juvenile* in 2 studies

Other Studies

2 other study(ies) available for sirolimus and Leukemia--Myelomonocytic--Juvenile

Article	Year
Control of thrombotic thrombocytopenic purpura by sirolimus in a child with juvenile myelomonocytic leukemia and somatic N-RAS mutation. Pediatric blood & cancer, 2014, Volume: 61, Issue:10 We describe an infant who developed juvenile myelomonocytic leukemia (JMML) at the age of 6 months. Myeloproliferation was effectively controlled by low-dose cytosine arabinoside and 13-cis retinoic acid therapy. Two years after therapy for JMML was stopped, at the age of 5 years, the patient developed autoimmune thrombotic thrombocytopenic purpura (TTP). TTP was transiently controlled by plasma exchange, prednisolone, rituximab, and cyclophosphamide, but relapsed within a short time. Long-term control of TTP was established by sirolimus. Somatic N-RAS G38A→Gly13Asp substitution was restricted to hematopoietic cells. The somatic N-RAS mutation may link myeloproliferation and autoimmunity. Topics: Age of Onset; Antibiotics, Antineoplastic; Antineoplastic Combined Chemotherapy Protocols; Child, Preschool; Cytarabine; Genes, ras; Humans; Infant; Isotretinoin; Leukemia, Myelomonocytic, Juvenile; Mutation; Purpura, Thrombotic Thrombocytopenic; Sirolimus	2014
PTEN deficiency is a common defect in juvenile myelomonocytic leukemia. Leukemia research, 2009, Volume: 33, Issue:5 The biological hallmark of juvenile myelomonocytic leukemia (JMML) is selective GM-CSF hypersensitivity. We hypothesized that PTEN protein deficiency might lead to insufficient negative growth signals to counter the hyperactive Ras signaling and therefore aid in the acceleration of the malignant transformation of JMML. In screening 34 JMML patients we found: (1) decreased PTEN protein in 67% of patients; (2) significantly lower PTEN mRNA levels in patients compared to controls (p<0.01); (3) a hypermethylated PTEN promoter in 77% of patients; and (4) constitutive-hyperactive Akt and MAPK in 55% and 73% of patients, respectively. These findings suggest that PTEN deficiency is very common in JMML and is in part due to hypermethylation of the PTEN gene promoter. Topics: Base Sequence; Blotting, Western; DNA Methylation; DNA Primers; Granulocyte-Macrophage Colony-Stimulating Factor; Humans; Leukemia, Myelomonocytic, Juvenile; Promoter Regions, Genetic; Protein Kinases; PTEN Phosphohydrolase; Reverse Transcriptase Polymerase Chain Reaction; RNA, Messenger; Sirolimus; Transcription, Genetic	2009

Article

Year

Control of thrombotic thrombocytopenic purpura by sirolimus in a child with juvenile myelomonocytic leukemia and somatic N-RAS mutation.

Pediatric blood & cancer, 2014, Volume: 61, Issue:10

We describe an infant who developed juvenile myelomonocytic leukemia (JMML) at the age of 6 months. Myeloproliferation was effectively controlled by low-dose cytosine arabinoside and 13-cis retinoic acid therapy. Two years after therapy for JMML was stopped, at the age of 5 years, the patient developed autoimmune thrombotic thrombocytopenic purpura (TTP). TTP was transiently controlled by plasma exchange, prednisolone, rituximab, and cyclophosphamide, but relapsed within a short time. Long-term control of TTP was established by sirolimus. Somatic N-RAS G38A→Gly13Asp substitution was restricted to hematopoietic cells. The somatic N-RAS mutation may link myeloproliferation and autoimmunity.

Topics: Age of Onset; Antibiotics, Antineoplastic; Antineoplastic Combined Chemotherapy Protocols; Child, Preschool; Cytarabine; Genes, ras; Humans; Infant; Isotretinoin; Leukemia, Myelomonocytic, Juvenile; Mutation; Purpura, Thrombotic Thrombocytopenic; Sirolimus

2014

PTEN deficiency is a common defect in juvenile myelomonocytic leukemia.

Leukemia research, 2009, Volume: 33, Issue:5

The biological hallmark of juvenile myelomonocytic leukemia (JMML) is selective GM-CSF hypersensitivity. We hypothesized that PTEN protein deficiency might lead to insufficient negative growth signals to counter the hyperactive Ras signaling and therefore aid in the acceleration of the malignant transformation of JMML. In screening 34 JMML patients we found: (1) decreased PTEN protein in 67% of patients; (2) significantly lower PTEN mRNA levels in patients compared to controls (p<0.01); (3) a hypermethylated PTEN promoter in 77% of patients; and (4) constitutive-hyperactive Akt and MAPK in 55% and 73% of patients, respectively. These findings suggest that PTEN deficiency is very common in JMML and is in part due to hypermethylation of the PTEN gene promoter.

Topics: Base Sequence; Blotting, Western; DNA Methylation; DNA Primers; Granulocyte-Macrophage Colony-Stimulating Factor; Humans; Leukemia, Myelomonocytic, Juvenile; Promoter Regions, Genetic; Protein Kinases; PTEN Phosphohydrolase; Reverse Transcriptase Polymerase Chain Reaction; RNA, Messenger; Sirolimus; Transcription, Genetic

2009