Effectiveness of an educational health programme on mothers' knowledge and practices of thalassaemic children receive desferal therapy in Hawler Thalassemia Center/ Erbil city

Thalassaemia is a heredity blood diseases characterised by decreased synthesis of one of the two types of polypeptide chains [beta or alpha] which form the normal adult human hemoglobin molecule [HbA, alpha2,beta2], resulting in decreased filling of the red cells with haemoglobin, and cause anaemia. The study aimed to improve mothers' knowledge and practices of Thalassaemic children who are using Desferal therapy. A quasi-experimental study was carried out at Hawler Thalassemia Center in Erbil City from the 1[st] of March to the end of May 2010. One hundred mothers were selected and divided into two groups, 50 mothers exposed to the educational programme [study group] and a second group of [50] mothers were served as control. Pre and post test of subject of interest were done during the two occasions. The results revealed that mothers' knowledge and practices in the study group were improved. There is no significant association between mothers' knowledge and practices with socio-demographic characteristics at pre-test which became significant at post-test. Yet, most of the mothers in the study group have gained benefit from implementation of this educational programme

Past, present & future scenario of thalassaemic care & control in India.

The first case of thalassaemia, described in a non-Mediterranean person, was from India. Subsequently, cases of thalassaemia were documented in all parts of India. Centres for care of thalassaemics were started in the mid-1970s in Mumbai and Delhi, and then in other cities. The parent's associations, with the help of International Thalassemia Federation, greatly helped in improving the care of thalassaemics. Obtaining blood for transfusion was difficult, but the Indian Red Cross Society and the parent's associations played a crucial role in arranging voluntary donations of blood. Chelation with deferoxamine was used sparingly due to the high cost. The Indian physicians conducted trials with deferiprone, and the drug was first approved and marketed in India. Deferasirox is also now being administered. Studies of physical and pubertal growth documented significant retardation, suggesting that generally patients receive inadequate chelation and transfusions. Bone marrow transplantation is available at a number of centres, and cord blood stem cell storage facilities have been established. Information about mutations in different parts of India is available, and ThalInd, an Indian database has been set up. There is a need to set up preimplantation genetic diagnosis and non-invasive prenatal diagnosis. It is argued that too much emphasis should not be placed on premarital screening. The focus should be on screening pregnant women to yield immediate results in reducing the burden of this disorder. Care of thalassaemia has been included in the 12th 5-year Plan of the Government of India. Many States now provide blood transfusions and chelation free of cost. Although inadequacies in care of thalassaemia remain, but the outlook is bright, and the stage is set for initiating a control programme in the high risk States.

evaluation of sensory neural hearing loss in thalassaemic patients treated with desferrioxamine and its risk factors

In major thalassaemia patients who need blood transfusion, iron overload is a major therapeutic disadvantage that leads to heart failure which is the major cause of death in such patients. Desferrioxamine [DFO] is the most efficient factor for iron chelation, but it carries adverse effects such sensory-neural hearing loss. The study began in March 2002 and continued untill March 2003, on 160 cases of thalassaemia to determine the incidence of sensory - neural hearing loss and its risk factors in patients who received Desferrioxamine [DFO]. All cases underwent audiometric tests. Retrospectively, other needed information were either obtained through interview or extracted from the medical files. Results were analyzed with ANOVA, t-test and Chi-square tests. Seventy-six patients of the total 156 patients showed impairment in PTA [48.7%] with 24 of them suffering significant involvement [15.4%]. These abnormalities generally affected high frequencies including, 4000 and 8000 Hz. Male gender, increased serum billirubin level and fasting blood sugar were statistically correlated with hearing loss [p.v = 0.038, p.v = 0.38, p.v = 0.002 respectively]. There was no significant correlation between hearing loss and other factors. Mean DFO administration in patients, was 29.69 mg/kg/day and mean therapeutic index of DFO was 0.01 mg/kg/day/mg/lit. Both of them were below the critical level [<40mg/kg/day and <0.025mg/kg/day/mg/lit respectively],however hearing loss had developed. Controlling DFO dosage per se does not seem to be enough for decreasing ototoxicity rate. Periodic audiometric tests are highly recommended to detect hearing loss as soon as possible. There are some other factors such as male gender, increased billirubin and FBS, which contribute to DFO ototoxicity. Looking for these risk factors and controlling them, would help identifying susceptible patients and preventing this complication

Deferiprone, efficacy and safety.

OBJECTIVE: Deferiprone (L1), the new oral iron chelator has been studied in several countries for its efficacy and toxicity with some conflicting observations. Toxicity involving joints has been reported more frequently in Indian patients. The authors planned to include larger number of Indian thalassemics in studying safety and efficacy of Deferiprone. METHODS: Seventy five thalassemic children (4-14 yr) were studied for one year with various investigations done periodically. Thirty patients (group A) received 50 mg/kg dose and 21 others (group B) received 75 mg/kg dose of Deferiprone. Rest of the patients were followed up without any chelator. RESULTS: The serum ferritin levels reduced significantly in both groups (P < 0.01 each); more in 75 mg/kg than the 50 mg/kg group. Arthropathy appeared in 15 (50%) patients in Group A and 6 (28.6%) of Group B after 1-12 (mean 6) months of L1 treatment; however, only one patient needed withdrawal of L1. Eleven patients needed indomethacin for pain relief. Seropositivity for antinuclear factor and rheumatoid factor had no relation to dose or duration of L1 therapy, arthropathy or the serum ferritin level. Twelve patients developed leucopenia (< 3.0 x 10(9)/L) and neutropenia (0-1.8 x 10(9)/L) after 2-11 months of L1 therapy and was not related to the dose or duration of therapy. The drug was restarted in 10 patients and only one of them developed a second episode of neutropenia. CONCLUSION: Deferiprone is an effective iron chelator, but arthropathy and neutropenia are very frequent side effects and need strict monitoring during therapy. Most of the neutropenia are neither very severe nor recur with re-challenge with the drug. Similarly, arthropathy does not need withdrawal of drug in majority of patients.

Acute graft-versus-host disease in thalassaemic marrow transplantation with low-dose antithymocyte globulin

Our unit performed transplantations on 21 classes II and III thalassaemic patients [class II patients had either hepatomegaly or portal fibrosis and class III patients had both]. We used busulfan [15 mg/kg] and cyclophosphamide [200 mg/kg]. Graft-versus-host disease [GVHD] prophylaxis was cyclosporin, prednisolone and low-dose antithymocyte globulin. Our patient data showed a low incidence of acute GVHD following transplantation. We offer this regimen as an acceptable therapy for thalassaemic patients undergoing allogeneic marrow transplantation as a safe clinical procedure, irrespective of the class of patient

Minimal doses of hydroxyurea for sickle cell disease

The use of hydroxyurea (HU) can improve the clinical course of sickle cell disease. However, several features of HU treatment remain unclear, including the predictability of drug response and determination of adequate doses, considering positive responses and minimal side effects. In order to identify adequate doses of HU for treatment of sickle cell disease, 10 patients, 8 with sickle cell anemia and 2 with Sbeta thalassemia (8SS, 2SBeta), were studied for a period of 6 to 19 months in an open label dose escalation trial (10 to 20 mg kg(-1) day(-1)). Hemoglobin (Hb), fetal hemoglobin (Hb F) and mean corpuscular volume (MCV) values and reticulocyte, neutrophil and platelet counts were performed every two weeks during the increase of the HU dose and every 4 weeks when the maximum HU dose was established. Reduction in the number of vasoocclusive episodes was also considered in order to evaluate the efficiency of the treatment. The final Hb and Hb F concentrations, and MCV values were significantly higer than the initial values, while the final reticulocyte and neutrophil counts were significantly lower. There was an improvement in the concentration of Hb (range: 0.7-2.0 g/dl) at 15 mg HU kg(-1) day(-1), but this concentration did not increase significantly when the HU dose was raised to 20 mg kg(-1) day(-1). The concentration of Hb F increased significantly (range: 1.0-18.1 per cent) when 15 mg HU was used, and continued to increase when the dose was raised to 20 mg kg(-1) day (-1). The final MCV values increased 11-28 fl (femtoliters). However, reticulocyte (range: 51-205 x 10(9)/l) and neutrophil counts (range: 9.5-1.3 x 10(9)/l) obtained at this dose were significantly lower than those obtained with 15 mg kg(-1) day(-1). All patients reported a decrease in frequency or severity of vasoocclusive episodes. These results suggest that a hydroxyurea dose of 15 mg kg(-1) day(-1) seems to be adequate for treatment of sickle cell disease in view of the minimal side effects observed and the improvement in laboratory and clinical parameters.

Cytological effects desferrioxamine in somatic cells of Swiss albino mice

Desferroxamine an iron chelator, has been shown to be ineffective in increasing the frequency of micronuclei in bone marrow cells of mice on acute and sub - acute treatment. However, a significant mitodepressive effect is produced at the higher dose on sub - acute treatment. The mito - depressive potentials of DFO may be either due to the disproptionate ratio of Fe2+/Fe3+ which is known to stimulate lipid peroxidation or due to total chelation of iron resulting in bone narrow depression and anemic condition

Novel thrombolytic therapy discovered from traditional oriental medicine using the earthworm.

Since a few thousand years ago, the earthworm has been used as a drug for various diseases in China and the Far East. However, modern scientific pharmacological studies have not so far been performed. We extracted a very strong fibrinolytic enzyme from the earthworm, Lumbricus rubellus. This enzyme was heat-stable and displayed a very broad optimal pH range. Purification of the enzyme was performed and three partially purified fractions were obtained. These three fractions were further subdivided, and six purified fractions (F-I-0, 1, 2, F-II, and F-III-1,2) were finally obtained. Based on results of their enzymatic activities against various substrates, the fraction I enzymes are thought to represent chymotrypsin-like enzymes and the fraction III enzymes to represent trypsin-like enzymes. The fraction II enzyme appears to be neither a trypsin-nor chymotrypsin-like enzyme nor an elastase. We therefore designed trials for in vivo experiments on human volunteers. 120 mg of lyophilized earthworm powder was administered orally to 7 healthy volunteers (aged 28-52 years old) three times after meals every day for 17 days. Blood was withdrawn once a day before and at 1, 2, 3, 8, 11 and 17 days after commencing the administration. The fibrin degradation products (FDP) value, tissue plasminogen activator (t-PA) antigen level and t-PA activities were measured in the blood. Before the administration, the t-PA antigen level was 5.6 +/- 0.38 ng/ml, and it gradually increased until the 17th day. The FDP level was increased on the 1st and 2nd day after the administration, but had decreased and normalized by the 17th day. The fibrinolytic activities also tended to show an increase during the experiment. These results suggest that earthworm powder represents a possible oral thrombolytic agent. The earthworm enzyme may thus be applicable for treating patients with thalassemia.

Iron excretion in thalassemia children treated with deferoxamin methansulfonat: intravenous drip compared to the subcutaneous routes.

The iron excretion in the three beta-Thal/Hb E patients were determined comparing the effect of DF given by subcutaneous push, subcutaneous drip and intravenous drip. The subcutaneous drip or intravenous drip increased urine iron excretion by 5.6-11.2 times whereas the subcutaneous push, 3.5-5.3 times only. It is recommended that for countries where the infusion machine is very expensive the DF should be given by intravenous drip or the modified, simple and inexpensive equipment for subcutaneous drip.

Talasemia mayor: terapia quelante con deferoxamina / Thalassemia major: chalation therapy with deferoxamine

Para evaluar los resultados de un año de tratamiento con deferoxamina subcutánea continua lenta, cuya meta es alcanzar un balance negativo del hierro (Bal-Fe) (que se elimine más del que se incorpora con las transfusiones), se estudiaron 10 talasémicos mayores (6 varones y 4 mujeres, edad 2-17 años) sometidos a dicha terapia y bajo régimen de "hipertransfusión moderada" (además de suplementación con ácido fólico y vitamina C). Los depósitos de Fe se midieron dosando la ferritina sérica, la incorporación por el volumen de glóbulos desplasmatizados transfundidos en el lapso estudiado y la eliminación multiplicando la sideruria diaria promedio por el total de días que recibió el quelante (más un 25% correspondiente a la eliminación fecal, no determinada). Los resultados muestran que sólo 2 pacientes (2 niñas de 12 años) lograron Bal-Fe; en otros 5 la respuesta fue buena pero insuficiente y en los 3 restantes (todos de corta edad) la respuesta fue más limitada (depósitos aun escasos?). Se concluye que los resultados obtenidos son satisfactorios, pues con mayores dosis de deferoxamina y/o esplenectomía (que reduce el requirimiento transfusional) sería factible una negativización del balance en los que todavía no lo lograron; y en los de menor edad, no se descarta que la terapia retrase o prevenga una sobrecarga exagerada, hasta tanto el crecimiento permita la aplicación de las otras medidas (dosis superiores, esplenectoía). Pese a sus desventajas (alto costo, aceptación dificultosa), consideramos que momentáneamente constituye el tratamiento de elcción