Myelodysplastic syndromes with single neutropenia or thrombocytopenia are rarely refractory cytopenias with unilineage dysplasia by World Health Organization 2008 criteria and have favourable prognosis

Gyan, Emmanuel; Andrieu, Valérie; Sanna, Alessandro; Caille, Agnès; Schemenau, Jennifer; Sudaka, Isabelle; Siguret, Virginie; Malet, Michèle; Park, Sophie; Bron, Dominique; Mairesse, Jacques; Gelsi‐Boyer, Véronique; Chèze, Stéphane; Beyne‐Rauzy, Odile; Sébert, Marie; Sapena, Rosa; Zerazhi, Hacène; Legros, Laurence; Guerci‐Bresler, Agnès; Amé, Shanti; Germing, Ulrich; Salvi, Flavia; Gioia, Daniela; Lunghi, Monia; Dreyfus, François; Fenaux, Pierre

doi:10.1111/bjh.13902

Cited by 16 publications

(10 citation statements)

References 6 publications

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“…The SNFMI registry included 123 MDS/CMML patients with AID in 26 centres) [13], while the GFM registry included 4202 MDS/CMML in 30 centres of whom 80 also had documented AID. The GFM registry has been so far the basis for many publications of the GFM or that included the GFM [14][15][16].…”

Section: Methodsmentioning

confidence: 99%

Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia

et al. 2016

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This retrospective study describes efficacy of Azacitidine on autoimmune disorders (AID) associated with MDS/CMML in 22 patients. Response of AID to Azacitidine was observed in 19 patients (86%). Reduction or discontinuation of steroids and/or immunosuppressive therapy (IST) was possible in 16 cases (73%). Hematologic response was seen in 55% of the patients. MDS/CMML and AID evolution was concordant in 13 cases (59%): both favorable (n=11), both unfavorable (n=2), but AID improved while MDS/CMML worsened (n=8) and vice versa (n=1). Azacitidine frequently seems effective in controlling steroid-dependent AID associated with MDS/CMML, but prospective studies are necessary to confirm those findings.

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Section: Methodsmentioning

confidence: 99%

Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia

et al. 2016

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“…In addition, clinical trials clearly show that the percentage of infectious events is high, not only in the therapeutic arm but also in the control group, emphasizing that there is an inherent risk of infection in MDS, regardless of the therapeutic approach, due to both neutropenia and the altered function of neutrophils [11][12][13][14][19][20][21]. When detailed, most reported infections in MDS occur in the presence of neutropenia and are prevalently of bacterial origin, with subsequent pneumonia, bacteremia and/or sepsis [4,28,29] Although infections may occur and cause death independently of specific risk factors [10,[28][29][30][31][32][33][34][35], baseline neutropenia, present in > 50% of higher-risk IPSS/IPSS-R and 15-20% of lower-risk MDS, is likely the main predisposing factor in these patients [4,6,29,35]. Furthermore, several other functional defects of granulocytes, as well as various types of B-, T-, natural killer and regulatory T-cell abnormalities, have been reported to also impair the response to infectious microorganisms in the absence of neutropenia in patients with MDS [4,31].…”

Section: Epidemiology Of Infections In Patients With Mds Receiving Current Therapymentioning

confidence: 99%

Infection control in patients with myelodysplastic syndromes who are candidates for active treatment: Expert panel consensus-based recommendations

et al. 2019

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The improvement in supportive care and the introduction of new therapeutic agents, including lenalidomide and hypomethylating agents, in myelodysplastic syndromes have improved patients' outcomes; however, at the same time, the frequency and epidemiology of infections have changed. Therefore, the great strides in the indications and use of new treatment strategies for myelodysplastic syndromes need a parallel progress in the best approach to prophylaxis and supportive therapy for infections. Based on the recognition that the above issues represent an unmet clinical need in myelodysplastic syndromes, an Italian expert panel performed a review of the literature and composed a framework of the best recommendations for optimal infection control in patient candidates to receive active treatment for myelodysplastic syndromes. In this consensus document we report the outcomes of that review and of the consensus meetings held during 2017. The issues tackled in the project dealt with: information to be collected from candidates for active treatment for myelodysplastic syndromes; how to monitor the risk of infection; antimicrobial prophylaxis; the role of iron chelation and antiviral/antibacterial vaccinations. For each of these issues, practice recommendations are provided.

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“…Although THC2 is rare disease, isolated thrombocytopenia and BM dysplasia limited to the megakaryocyte lineage are very rare 22 , 23 and carry a higher likelihood of progression to MN. Thus, when dysmegakaryopoiesis is found in a patient having thrombocytopenia, the differential diagnosis of MDS and hereditary thrombocytopenia should be investigated.…”

Section: Resultsmentioning

confidence: 99%

Inherited Thrombocytopenia Caused by Germline ANKRD26 Mutation Should Be Considered in Young Patients With Suspected Myelodysplastic Syndrome

Kewan

Noss

Godley

et al. 2020

Journal of Investigative Medicine High Impact Case Reports

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Thrombocytopenia 2 (THC2) is an autosomal dominant disorder characterized by ankyrin repeat domain 26 mutation and moderate thrombocytopenia. THC2 exposes patients to a low risk of bleeding and an increased likelihood of myelodysplastic syndrome/acute myeloid leukemia. Germline evaluation for a genetic disorder should be considered when a patient presents with isolated thrombocytopenia and associated dysmegakaryopoiesis. In this case report, we present a male patient who presented with isolated thrombocytopenia but was ultimately confirmed to have an inherited THC2 thrombocytopenia/myelodysplastic syndrome. Given the rarity of the disease, no clear guidelines on how to follow THC2 patients over the long term have been established. We recommend a monthly complete blood count and clinical visits every 3 months at a minimum.

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Myelodysplastic syndromes with single neutropenia or thrombocytopenia are rarely refractory cytopenias with unilineage dysplasia by World Health Organization 2008 criteria and have favourable prognosis

Cited by 16 publications

References 6 publications

Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia

Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia

Infection control in patients with myelodysplastic syndromes who are candidates for active treatment: Expert panel consensus-based recommendations

Inherited Thrombocytopenia Caused by Germline ANKRD26 Mutation Should Be Considered in Young Patients With Suspected Myelodysplastic Syndrome

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