Complications and treatment of patients with  -thalassemia in France: results of the National Registry

Thuret, Isabelle; Pondarré, Corinne; Loundou, Anderson; Steschenko, Dominique; Girot, Robert; Bachir, Dora; Rosé, Christian; Barlogis, Vincent; Donadieu, Jean; Montalembert, Mariane de; Hagège, Isabelle; Pégourie, Brigitte; Berger, Claire; Micheau, M; Bernaudin, Françoise; Leblanc, Thierry; Lutz, Laurence; Galactéros, F; Siméoni, Marie-Claude; Badens, Catherine

doi:10.3324/haematol.2009.018051

Cited by 88 publications

(105 citation statements)

References 15 publications

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“…Observation of nontransplanted thalassemia major patients in the national registry of France indicates that better-managed patients may have lower serum ferritin levels (1240 ng/mL). 10 This excess iron store, as shown by increased ferritin values, seems to mediate several of the complications of thalassemia. In well-transfused patients, poor compliance to chelation treatment and subsequent iron overload remain the main causes of poor growth.…”

Section: Discussionmentioning

confidence: 99%

Physical growth in children with transfusion-dependent thalassemia

Pemde¹,

Chandra²,

Gupta³

et al. 2011

PHMT

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Objective: To describe physical growth and related factors in transfusion-dependent thalassemia patients. Methods: This is a cross-sectional analysis of the records of the patients registered at and being followed up by the Thalassemia Day Care Center (TDCC) at Kalawati Saran Children's Hospital, New Delhi, India. Clinical and laboratory parameters were recorded on a spreadsheet for analysis. Clinical parameters included weight, height, sexual maturity ratings, and general and systemic physical examination. Laboratory parameters included pretransfusion hemoglobin (Hb), periodic serum ferritin, and tests for viral markers of human immunodeficiency virus (HIV) and hepatitis B and C. Z-scores for weight, height, and body mass index (BMI) were calculated using World Health Organization reference data. Statistical analysis was carried out using Microsoft Excel ® and Stata ® software. Results: Out of 214 patients registered at the TDCC since 2001, 154 were included in this study. The mean age of patients was 9.19 years (range 0.5-20 years). Pretransfusion Hb was well maintained (mean 9.21 g/dL; 95% confidence interval [CI]: 9.06-9.36), but the mean serum ferritin levels were approximately three times (3112 ng/mL) the desired value despite the patients being on deferiprone (72%) or deferasirox (25%). One-third (33.11%) of the patients had short stature, 13% were thin, and 10.82% were very thin (BMI z-score ,-3). No patient was overweight or obese. Linear regression coefficient showed that for every 1-year increase in age, the mean ferritin value increased by 186.21 pg/mL (95% CI: 143.31-228.27). Height z-scores had significant correlation with mean ferritin levels, whereas correlation with mean pretransfusion Hb was not significant statistically. Mean ferritin levels were significantly higher in patients with short stature than in the patients with normal height. Regression analysis showed that an increase of 3571 units of serum ferritin was associated with a decrease of one point in height z-scores. One-fifth (19.40%) of adolescent patients had delayed puberty. Conclusion: Approximately one-third (33.11%) of patients with transfusion-dependent thalassemia major were of short stature. In this group of patients with pretransfusion Hb levels maintained at desired levels, physical growth was correlated with status of iron overload.

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Section: Discussionmentioning

confidence: 99%

Physical growth in children with transfusion-dependent thalassemia

Pemde¹,

Chandra²,

Gupta³

et al. 2011

PHMT

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“…Clinical data were collected retrospectively on all 922 thalassemia patients in cohort C. Diagnosis of thalassemia was made through a comprehensive assessment of clinical presentation and hematological and molecular analysis based on criteria previously described. 23,24 A summary of clinical and genetic characterizations of 922 patients, 568 thalassemia major (TM) and 354 thalassemia intermedia (TI) in cohort C, is presented in Table 1.…”

Section: Population Patients and Phenotypic Testsmentioning

confidence: 99%

KLF1 mutations are relatively more common in a thalassemia endemic region and ameliorate the severity of β-thalassemia

Liu

Zhang

et al. 2014

Blood

156

124

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“…It has been postulated that post-splenectomy conformational changes in RBC membranes enhance immunomodulation that result in allosensitization. While splenectomy is performed less frequently in thalassaemia [6,36], the significantly increased risk of alloimmunization in splenectomized younger patients is concerning and deserves further study.…”

Section: Other Factorsmentioning

confidence: 99%

Red Blood Cell Antibodies in Thalassemia Patients in Northern India: Risk Factors and Literature Review

Elhence

Solanki

Verma

2014

Indian J Hematol Blood Transfus

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Study of the factors responsible for red cell alloimmunization can help in adopting appropriate strategy to minimize alloimmunization. However data for thalassemia patients from our region is limited. Therefore, a study was conducted to find out the frequency and the factors associated with red cell allo and autoimmunization in thalassemia patients at our center so as to enable us to take appropriate action to reduce alloimmunization. Clinical, demographic, allo and autoantibody and transfusion records of 280 thalassemia patients at our hospital were studied. Patients with and without alloantibodies were compared to find significant differences for age, gender, race, age at start of regular transfusions and splenectomy. Red cell antigen frequencies in thalassemia patients and published antigen frequencies in blood donors from the same center were compared to look antigen differences as a risk factor for alloimmunization. Twenty four thalassemia patients (8.6 %) developed 28 clinically significant alloantibodies. 18 (65 %) of the alloantibodies were of Rh system. The three most common antibodies detected was anti E (11, 39.3 %) followed by anti K (6, 21.4 %) and anti c (10.8 %). Five (1.8 %) of the 280 patients developed autoantibodies. Patient age was found to be significantly higher in alloimmunized patients than in non alloimmunized patients. Red cell antigen frequencies between blood donor and recipient populations were found to be homogenous for most of the relevant RBC antigens. The frequency of red cell alloimmunization in thalassemia patients from our center is moderate. In this setting of red cell phenotype concordant donor-recipient population requirement of extended phenotype matched transfusions may not be cost effective.

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Complications and treatment of patients with -thalassemia in France: results of the National Registry

Cited by 88 publications

References 15 publications

Physical growth in children with transfusion-dependent thalassemia

Physical growth in children with transfusion-dependent thalassemia

KLF1 mutations are relatively more common in a thalassemia endemic region and ameliorate the severity of β-thalassemia

Red Blood Cell Antibodies in Thalassemia Patients in Northern India: Risk Factors and Literature Review

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