3.Research Progress of Thalidomide and Its Derivatives in Treatment of Myelodysplastic Syndrome--Review.
Jing-Wen WANG YIN ; Gao-Feng JIANG ; Jian NI ; Yong-Ming ZHOU
Journal of Experimental Hematology 2021;29(6):1967-1971
Thalidomide and its derivatives have been used in the treatment of myelodysplastic syndrome (MDS) because of their anti-angiogenic and immunomodulatory effects. In recent years, some studies have found that thalidomide and its derivatives not only showed significant efficacy in lower-risk MDS patients with del (5q), but also showed advantages in non-del (5q) MDS patients. In addition, the discovery of its molecular targets and new substrates makes it possible to develop a new generation of immunomodulatory drugs (IMiDs) and to design IMiDs-based proteolysis-targeting chimeras. In this review, the new progress in mechanism and clinical application of thalidomide and its derivatives were summarized briefly, so as to provide a more scientific, reasonable and effective scheme to the treatment of MDS.
Humans
;
Immunomodulating Agents
;
Myelodysplastic Syndromes/drug therapy*
;
Thalidomide/therapeutic use*
4.A Case of Disseminated Trichosporon beigelii Infection in a Patient with Myelodysplastic Syndrome after Chemotherapy.
Jong Chul KIM ; Yang Soo KIM ; Chul Sung PARK ; Jae Myung KANG ; Baek Nam KIM ; Jun Hee WOO ; Jiso RYU ; Woo Gun KIM
Journal of Korean Medical Science 2001;16(4):505-508
Trichosporonosis is a potentially life-threatening infection with Trichosporon beigelii, the causative agent of white piedra. The systemic infection by this fungus has been most frequently described in immunocompromised hosts with neutropenia. Here, we report the first patient with disseminated infection by T. beigelii in Korea, acquired during a period of severe neutropenia after chemo-therapy for myelodysplastic syndrome. The patient recovered from the infection after an early-intensified treatment with amphotericin B and a rapid neutrophil recovery. The disseminated infection by T. beigelii is still rare, however, is an emerging fatal mycosis in immunocompromised patients with severe neutropenia.
Adult
;
Amphotericin B/therapeutic use
;
Human
;
Male
;
Mycoses/drug therapy/*etiology
;
Myelodysplastic Syndromes/*complications/drug therapy
6.Histone deacetylase inhibitors for the treatment of myelodysplastic syndrome.
Ya-Qin ZHI ; Shan-Qi GUO ; Yi-Zhuo ZHANG
Journal of Experimental Hematology 2012;20(3):792-795
The effects of conventional treatment for myelodysplastic syndrome (MDS) are not remarkable to date, while only a minority of patients was eligible for allogeneic stem cell transplantation. As epigenetics plays a significant role during the occurrence and development of MDS, and histone deacetylase inhibitors (HDACI), a class of gene expression modulating drugs, are currently being developed for therapy of several types of solid tumor, more attention is paying to HDACI as potential therapy of MDS. This review summarizes briefly the rationale for HDACI use in MDS, the common mechanism of HDACI, the present state of the clinical efficiency, and future development in this field.
Epigenesis, Genetic
;
Histone Deacetylase Inhibitors
;
therapeutic use
;
Humans
;
Myelodysplastic Syndromes
;
drug therapy
;
genetics
7.Application of cyclosporine A in myelodysplastic syndrome--review.
Journal of Experimental Hematology 2003;11(6):678-680
Treatment of myelodysplastic syndrome (MDS) remains unsatisfactory. It is possible that immunosuppressive therapy might be effective for a certain subset of patients with MDS. In this review 105 patients with MDS who were treated with cyclosporin A (CsA) including 90 RA, 5 RARS, 10 RAEB, were analyzed. The dose of CsA was 2 - 12 mg/(kg x d) for at least three months. Hematological improvement was observed in 64 patients (61%), and complete remission was observed in 14 patients (13.3%). These results indicated that CsA immunosuppressive therapy may be useful for IPSS low, intermediate-1 and intermediate-2 MDS patients.
Cyclosporine
;
adverse effects
;
therapeutic use
;
Humans
;
Immunosuppressive Agents
;
therapeutic use
;
Myelodysplastic Syndromes
;
drug therapy
;
immunology
8.Clinical Efficacy and Prognostic Factors of Decitabine for Treatment of Myelodysplastic Syndrome.
Zhen-Hua BAO ; Hong-Guo ZHAO ; Hong-E YU
Journal of Experimental Hematology 2018;26(6):1702-1707
OBJECTIVE:
To explore the clinical efficacy of decitabine for treatment of patients with myelodysplastic syndrome (MDS) and factors predicting the prognosis.
METHODS:
The clinical data of 87 patients with MDS treated with decitabine were analyzed retrospectively. The hENT1 mRNA expression and TP53 gene mutation were detected by Q-PCR and gene target sequencing, respectively. The relationship of clinical characteristics and molecular indicators with the clinical response to decitabine was analyzed.
RESULTS:
In treatment for median 4 (2-17) courses, a total 51 patients (58.6%) showed therapeutic responses, including CR in 17 cases, PR in 12 cases, mCR in 9 cases, HI in 13 cases; 36 (41.4%) patients showed non-response. Univariate analysis showed that the patients with the complex karyotype, monosomal karyotype, chomosome 7 abnormality and Plt count doubling after 1 course treatment had a high CR rate, while the patients with relative high risk by IPSS (intermediate risk 2+ high risk), complex karyotype and Plt count doubling after 1 course had much more high overall remission rate (ORR). The expression level of hENT1 mRNA in MDS patients with response was significantly higher than that in patients without response [(1.78±1.45 (2) vs 0.96±0.97 (2)(P= 0.002)]. Among 51 patients with therapeutic response, the expression level of hENT1 mRNA in CR group was higher than that in non-CR group [(2.58±1.44 (2) vs 1.39 ±1.3 (2), P= 0.005)]. Among 52 patients in relative high risk (intermediate risk 2 +high risk), the median OS time of patients with high hENT1 mRNA expression was significantly longer than that of patients with low hENT1 mRNA expression (31 vs 12 months)(P<0.001). Among 87 patients received decitabine treatment, the TP53 gene mutation occured in 11 (12.6%) patients. The ORR in patients with TP53 mutation was high (P=0.04), moreover the patients with TP53 mutation more easily gained CR (P<0.001). Multivariate logistic regression model showed that the complex karyotype, Plt count doubling after 1 course treatment, TP53 mulation and high expression of hENT1 mRNA were the independent prognostic factors for predicting the CR after decitabine treatment.
CONCLUSION
IPSS staging, complex karyotype, Plt count doubling after 1 course treatment and hENT1 mRNA expression, TP53 gene mutation can be used to predict the tharapeutic efficacy of dectitabine for treatment of MDS.
Decitabine
;
therapeutic use
;
Humans
;
Myelodysplastic Syndromes
;
drug therapy
;
Prognosis
;
Retrospective Studies
;
Treatment Outcome
9.Treatment and clinical outcomes of patients relapsing after allogeneic hematopoietic cell transplantation for myelodysplastic syndrome.
Eun Ji CHOI ; Je Hwan LEE ; Jung Hee LEE ; Han Seung PARK ; Sun Hye KO ; Miee SEOL ; Young Shin LEE ; Young Ah KANG ; Mijin JEON ; Kyoo Hyung LEE
Blood Research 2018;53(4):288-293
BACKGROUND: Although allogeneic hematopoietic cell transplantation (HCT) is the only curative treatment option for myelodysplastic syndrome (MDS), a substantial number of patients experience relapse. We reviewed the clinical outcomes of patients with MDS who relapsed after allogeneic HCT. METHODS: Thirty patients who experienced relapse or progression after allogeneic HCT for MDS between July 2000 and May 2016 were included in this retrospective analysis. RESULTS: The median time from HCT to relapse was 6.6 (range, 0.9–136.3) months. Donor lymphocyte infusions (DLIs) were administered to four patients: one achieved complete remission (CR) and survived disease free, while three did not respond to DLI and died. Hypomethylating agents were administered to seven patients: one who had stable disease continuously received decitabine, while six died without response to treatment. Six patients received AML-like intensive chemotherapy, and three achieved CR: two underwent second HCT and one DLI. One patient receiving second HCT survived without disease, but the other two relapsed and died. Three, four, and eight patients who did not respond to intensive chemotherapy, low-dose cytarabine, and best supportive care, respectively, died. One patient who underwent second HCT following cytogenetic relapse survived disease free. Median overall survival after relapse was 4.4 months, and relapse within 6 months after HCT was associated with shorter survival. CONCLUSION: Outcomes of MDS patients relapsing after allogeneic HCT were disappointing. Some patients could be saved using DLI or second HCT.
Cell Transplantation*
;
Cytarabine
;
Cytogenetics
;
Drug Therapy
;
Humans
;
Lymphocytes
;
Myelodysplastic Syndromes*
;
Recurrence
;
Retrospective Studies
;
Tissue Donors
;
Transplants*
10.Fatal Breakthrough Mucormycosis in an Acute Myelogenous Leukemia Patient while on Posaconazole Prophylaxis.
Seung Hun KANG ; Hyun Seon KIM ; Myoung Nam BAE ; Jihye KIM ; Ji Yeon YOO ; Kwan Yong LEE ; Dong Gun LEE ; Hee Je KIM
Infection and Chemotherapy 2015;47(1):49-54
Posaconazole is a new oral triazole with broad-spectrum antifungal activity. Posaconazole has also shown a significant advantage of preventing invasive fungal infection compared to fluconazole or itraconazole in patients with prolonged neutropenia. Indeed, posaconazole has been commonly used for antifungal prophylaxis in patients undergoing remission induction chemotherapy for acute myelogenous leukemia or myelodysplastic syndrome. We experienced a case of fatal mucormycosis despite posaconazole prophylaxis. To our knowledge, this is the first reported case of fatal breakthrough mucormycosis in a patient receiving posaconazole prophylaxis during remission induction chemotherapy in Korea. This case demonstrated that breakthrough fungal infection can occurs in patients receiving posaconazole prophylaxis because of its limited activity against some mucorales.
Drug Therapy
;
Fluconazole
;
Humans
;
Itraconazole
;
Korea
;
Leukemia, Myeloid, Acute*
;
Mucorales
;
Mucormycosis*
;
Myelodysplastic Syndromes
;
Neutropenia
;
Remission Induction