Comparision of Allogeneic Hematopoietic Stem Cell Transplantation between Children with Thalassemia of Different Ages / 中国实验血液学杂志

OBJECTIVE@#To investigate the difference of therapeutic effects on children with thalassemia at different age after hematopoietic stem cell transplantation.@*METHODS@#The clinical data of children with thalassemia treated in our hospital were retrospectively analyzed. The children were divided into 2-5 years old group and 6-12 years old group. The success rate of implantation, transplant-related mortality, GVHD incidence, and other transplant-related complications, as well as thalassemia-free survival (TFS) were compared between the two groups.@*RESULTS@#The incidence of GVHD, hemorrhagic cystitis and severe oral mucositis after transplantation in the 2-5 years old group were significantly lower than those in the 6-12 years old group, while there was no statistically significant difference in the TFS between the two groups.@*CONCLUSION@#Children in the low age (2-5 years old) group show fewer complications and higher quality of life after transplantation, therefore, stem cell transplantation at 2-5 years old is more conducive to rehabilitation of the children with thalassemia.

The Clinical Efficacy of Haploidentical Hematopoietic Stem Cell Transplantation by Using Parental Donors in Patients with Thalassemia / 中国实验血液学杂志

OBJECTIVE@#To analyze the clinical efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) by using parental donors on thalassemia patients.@*METHODS@#The 13 thalassemia patients treated by haplo-HSCT using parental donors in our hospital from July 1, 2016, to July 1, 2020 were retrospectively reviewed. Hematopoiesis reconstitution, the incidence of GVHD, infections and the long-term survival of the patients were analyzed.@*RESULTS@#Twelve of the 13 patients were successfully implanted, the success rate of implantation was 92.3%. The median time of neutrophil and platelet engraftment was 12.5 days (range, 9-22 days) and 21 days (range,12-34 days), respectively. One patient achieved primary graft failure. Three (25%) patients developed to acute GVHD (aGVHD) and achieved complete remission after treatment. Chronic GVHD developed in three (25%) patients, one of them was extensive and under treatment, while one patient developed to severe bacterial infection (7.7%). CMV viremia was diagnosed in two patients (15.4%). There were no patients developed to CMV disease. Three (23.1%) patients achieved EB viremia after transplantation, one of them developed to EBV-related lymphocytic proliferative disease, while there were no patients showed invasive fungal infection. At the last follow-up, all patients survived, twelve of them were free from transfusion dependency. There were no transplant-related deaths. Projected overall and thalassemia-free survival at three years was 100% and 92.3%, respectively.@*CONCLUSION@#The transplant protocol of haplo-HSCT by using parental donors in patients with thalassemia has reliable source of donors, high incidence of successful implantation and low incidence of GVHD, which can be used as an effective way to increase the source of donors in children with thalassemia.

Prevalence, Geographical Distribution, Social Problems and Treatment of Thalassemia Patients.

A total of 700 beta thalassemia patients were gathered at the outdoor of the Hematology department of Medical College, Kolkata, attending during the period from January 2010 to December 2010. The data is generated by the interaction between them and their family members in presence of the physicians. Actual ethnic identities, place of residence, parity and sibship together with social stigma, if any, faced by the patient or by the member of the family and details of their treatment including transfusion history were noted down and reconfirmed from past records. The prevalence of thalassemia was found 70% among Hindus of which the 62.26% was contributed by castes like Maishya, Namasudra, Barga Kshatriya and Panda Kshatriya. The relative prevalence of subtypes of b-thalassemia was found to be 65% E-b thalassemia, 30% Homozygous b-thalassemia and 5% S-b thalassemia. The aboard of the 85.28% patients were found to be localised along banks river Hooghly and Ganga delta region and we can thus consider tentatively that this belt is a thalassemia zone in West Bengal. The maximum number of cases showed parity to be 1 and there was considerable amount of reproductive wastage. Regarding social stigma, 82.71% responded with no stigma. Early diagnosis and early onset of transfusion with chelation therapy was found to provide benefit for the patient reducing the total amount of transfusion needed per year and also restoring the quality of their life.

Evaluation of osteopathy in thalassemia by bone mineral densitometry and biochemical indices.

Objective To evaluate osteopathy in thalassemia by bone mineral densitometry (BMD) and biochemical indices. Methods Prospective review analysis with no follow up from 2006 to 2007 of 42 regularly transfused thalassemics aged 10–25 years (27 boys, 15 girls) was done. Anthropometry, pubertal stage and symptomatology were noted. Urinary C–terminal cross–linked telopeptide of type–1 collagen (Crosslaps) by ELISA; serum 25–OH vitamin D and osteocalcin by RIA; parathyroid hormone (PTH) and ferritin by chemiluminescence and IGF–1 by Enzyme immunoassay were evaluated. Dual Energy X-ray Absorptiometry (DEXA) of lumbar spine and femur was done on Lunar prodigy system. Data was entered and analyzed using the SPSS for Windows software. Mean comparisons were done by ANOVA 1 and data was compared using Chi–square test and p value<0.05 was taken as significant. Results Of 42 patients, 81% had osteoporosis by Z–score of DEXA. Urinary crosslaps was high in 55%; 36% had increased osteocalcin; 62% had low vitamin D levels; 38% had high parathyroid levels and IGF–1 was low in 52%. Mean serum ferritin level was 5344±2855 ng/dl. There was statistical significance (p=0.046) between chronological age and BMD. All 42 cases were divided into two groups: Group–1 (Normal DEXA), Group–2 (Abnormal DEXA) and analysis of biochemical indices between two groups showed no significant difference in any of the biochemical parameters. Conclusion This study revealed majority of thalassemics with inadequate chelation have bone resorption with advancing chronological age and BMD should be evaluated regularly for early diagnosis to prevent morbidity.

Expenditure to treat thalassaemia: an experience at a tertiary care hospital in India

The medical and economic problem of thalassaemia are considered to be a vast public health problem in the thalassaemia belt countries, emphasizing more on prenatal diagnosis as the solution of the problem. A cross-sectional descriptive study was conducted in the Institute of Haematology and Transfusion Medicine located in Medical College, Kolkata, India to assess the socio-demographic profile, clinical presentation, expenditure for treatment of thalassaemia patients and awareness about cause and prevention of the disease. Thalassaemia patients attended the Govt. setting were mostly from lower socioeconomic status with low level of literacy. Annual expenditure for treatment of thalassaemia ranged from $ 108 to 432; depending on type of treatment with average cost per transfusion was $ 5.2 +/- 2.2. Average 18.5% +/- 14.3 of the total annual income was spent on the treatment for thalassaemia. Average man days or school days lost for the patients was 29.87 +/- 18.5 and 19.07 +/- 12.7 for the accompanying persons. Blood transfusion and carrier screening facilities should be decentralized to decrease the expenditure for treatment and alleviate the harassment of the families. Folate and calcium tablets, hepatitis B vaccination can be made available at government setting free of cost

Advances in management of thalassemia.

Thalassemias represent the most common single-gene disorder causing a major public health problem in India. Thalassemia and hemoglobinopathies probably developed over 7000 years ago as a defense against malaria. In simple terms, thalassemia is caused by a mutation in either the â-globin chain or the á-globin chain which combine equally in red cells to form hemoglobin. These mutations lead to varying degree of anemia resulting into thalassemia minor, intermedia or major. Present write up relates to advances in the management of â-thalassemia major.

Bone marrow transplantation in thalassemia [Part 1]

During the last two decades conventional therapy has improved the prognosis of thalassemia. However, despite such improvement it still remains a progressive disease with treatment-related complications such as hepatitis, liver fibrosis, and cardiac disease. Bone marrow transplantation [BMT] can prevent or delay progression of the aforementioned complications. The importance of clinical research in the field of BMT was recognized with the award of the 1990 Nobel Prize in Physiology and Medicine to E. Donnall Thomas, one of the pioneers of BMT in humans. George Mathe' was a pioneer in the early development of clinical BMT. Mathe' et al. were the first to describe graft-versus-host-disease [GVHD] and its treatment, and the graft-versus- leukemia [GVL] effect in human. The first BMT for beta-thalassemia major was performed successfully by Thomas et al. in Seattle, in 1981. In the same year another patient with beta-thalassemia major underwent BMT in Pesaro, Italy, by Lucarelli et al. Since then, several hundred transplantations have been performed worldwide, the majority of these in Italy. From 1991 through 2007 BMT have been performed on 497 [Tehran=342, Shiraz=155] blood transfusion dependent patients with thalassemia major in Iran, with disease-free survival of 71-77% respectively. Due to high graft failure and GVHD rates, BMT from alternative donors should be restricted to patients who have poor life expectancies because they cannot receive adequate conventional treatment or because of alloimmunization to minor blood antigens. Beginning in the early 1980s, it was shown that umbilical cord blood contained high levels of hematopoietic progenitor cells

Umbilical cord blood transplantation: newer trends.

During last ten years, over 4000 umbilical cord blood transplantations have been performed worldwide. The interest in this modality of transplantation has been growing as this provides easy access to an alternative source of stem cells for treating cancer and serious genetic disorders with otherwise fatal outcome or immense morbidity. Umbilical cord blood is a commonly discarded source of useful stem cells. The outcome of transplantation using cells from this source in children mirrors the results of unrelated donor transplantation and hence the procedure is widely accepted by paediatric transplant community. Results are, however, hampered in adults due to low cell dose. Newer techniques, such as pooled or sequential cord blood transplantation, may help to increase progenitor cell numbers and improve immune reconstitution. In near future, non-haematopoietic uses will make this even more exiting area. In this write-up, we will review this treatment including cord blood banking issues and the ethical concerns. We will discuss both paediatric and adult transplantations including certain new indications.

Prevalence of anti HCV, HBsAg and HIV antibodies in high risk recipients of blood and blood products.

Along with hepatitis B virus (HBV) and human immunodeficiency virus (HIV), Hepatitis C virus (HCV) is emerging as a major transfusion hazard. 22 cases of haemophilia (A 19, B 3) and 20 cases of thalassaemia (2 16, E(2) 4) constituted the study group. Patients tested for anti HCV (using third generation ELISA), HBsAg and antibodies to HIV I and II. Prevalence of anti HCV was 54.5% in haemophilics and 5% in thalassaemics. HBsAg was detected in 9.09% haemophilics and 5% thalassaemics. No anti HIV was detected in this cohort. Anti HCV seropositivity in haemophilics has increased compare to previous studies.

Experience with the oral iron chelator deferiprone in transfusion-dependent children.

OBJECTIVE: To establish efficacy and safety of deferiprone. DESIGN: Prospective study. SETTING: The Lady Ridgeway Hospital for Children, Colombo. PATIENTS: Transfusion-dependent children in the age group 1 to 15 years. INTERVENTION: Patients were given 75 mg/kg/day of deferiprone orally in divided doses. MEASUREMENTS: Efficacy of deferiprone therapy was assessed by 4 to 6 monthly serum ferritin (SF) assays. Safety of therapy was assessed by 4-weekly white cell counts and serum alanine aminotransferase (ALT) levels. The Z-score was used to assess the significance of the difference between the mean initial and final SF level. RESULTS: 82 patients received deferiprone therapy for a mean duration of 30 +/- 14 months. Initial SF levels ranged from 1115 to 12,165 micrograms/l with a mean of 5156 +/- 2631 micrograms/l. Final SF levels ranged from 312 to 15,285 micrograms/l with a mean of 2809 +/- 2380 micrograms/l (Z score 5.99; p < 0.001). Two (2.4%) children developed agranulocytosis which reverted to normal on discontinuation of treatment. 41 (50%) developed arthropathy and in 17 this was severe enough to require discontinuation of therapy. Serum ALT levels were raised in 35 (43%) patients but reverted to pretreatment values or lower despite continuation of deferiprone therapy. There was one death in a 9-year old child who developed diabetes mellitus and heart failure despite deferiprone therapy for 3 years. CONCLUSIONS: A final SF level < 2500 micrograms/l was achieved in 52% children. Severe arthropathy and agranulocytosis may necessitate permanent discontinuation of therapy.

Thalassemia syndromes in Saudi Arabia meta-analysis of local studies

The geographical distribution of Alpha and Beta-Thalassemias differ markedly. Alpha-Thalassemia being particularly prevalent in Southeast Asia and Beta-Thalassemia in the Mediterranean basin. Thalassemia syndromes are common in Saudi Arabia: the Beta-Thalassemia genes occur with variable frequency in different regions of Saudi Arabia and both B+ and Bo thalassemia have been reported. Alpha-Thalassemia is also highly prevalent here and the interaction with the sickle cell gene is commonly observed. Over the last few years, a great deal of information regarding the clinical, molecular and management of these disorders has accumulated in the literature. In this paper we summarize some of the recent studies on the subject along with our experience and our attempt to highlight a number of questions still awaiting answers

Dr. P. C. Sen Memorial Award Paper. A study of HIV infection in thalassaemia patients of rural Bengal.

Thalassaemia patients receiving repeated blood transfusions are vulnerable to transfusion related infections. HIV infection is the most life threatening of them all. Blood being the most efficient mode of transmission of HIV, increases the risk of infection even further. Although the National AIDS Control programme has laid down stringent rules regarding blood safety, it remained to be seen whether they were being followed meticulously especially in rural areas. The present study was conducted to identify the HIV status of multi-transfused thalassaemia patients attending hospital blood banks of rural Bengal. Only 3 (0.9%) of the 330 thalassaemia patients examined were found to be HIV positive. Although the situation has not reached alarming proportions, yet appropriate control measures must be adopted on a mass scale to prevent further spread of the world wide pandemic.

Diferencia de la concentración de hemoglobina en escolares afroecuatorianos y mestizos de la ciudad de Esmeraldas / Difference of the concentration of hemoglobina in afroecuatorianos and racially mixed students of the city of Emeralds

La diferencia en la concentración de hemoglobina y hematocrito entre blancos y negros ha sido motivo de controversia durante los últimos treinta años. Varios estudios han demostrado diferencias significativas que oscilan entre los 0.5 - 0.7 g/dl en los niveles de hemoglobina a favor de los individuos de raza blanca; lo que provocaría una sobre estimación del estado de anemia cercana al 10 por ciento. El establecer esta posible diferencia, permitirá la definición de puntos de corte diferenciales para su aplicación en encuestas nutricionales, manejo clínico, monitoreo de intervenciones dietéticas y de salud pública; especialmente en países como el nuestro, donde el 5 por ciento de la población total es de raza negra. Se seleccionaron propositivamente, mediante un diseño transversal 200 escolares de la ciudad de Esmeraldas, clínicamente sanos (100 afroecuatorianos y 100 mestizos), con edades comprendidas entre los 6 y 12 años de edad; quienes fueron sometidos a valoración antropométrica, análisis hematimétrico completo, determinación de protoporfirina eritrocitaria libre, examen coproparasitario, determinación indirecta de HbS y frotis sanguíneo para descartar la presencia de malaria. En la muestra general no se encontraron diferencias significativas entre la concentración de hemoglobina por grupo ...