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Table 1 Summary description of the selected studies excluding case reports

From: Hydroxyurea and blood transfusion therapy for Sickle cell disease in South Asia: inconsistent treatment of a neglected disease

Reference Study design Related findings Comments
Italia et al. [29] A prospective trial involving adult and paediatric homozygous sickle (Hb SS) and adult Hb S-β thalassaemia patients from Madhya Pradesh and Maharashtra of India Clinical severity scores were significantly reduced by HU therapy among all groups of patients with SCD (p < 0.001). After HU therapy 91% patients had no transfusion requirement Low dose HU therapy (10–15 mg/kg/day) showed impressive improvement in the clinical condition of Indian patients with SCD
Singh et al. [30] A prospective study involving SCA patients from Chhattisgarh, India Number of hospital admissions (p 0.03) and rate of crisis per year (p 0.008) was significantly reduced with HU therapy HU therapy significantly improved the acute clinical events related the SCA and increased the time interval between transfusions
Patel et al. [31] A prospective open label observational study involving SCA patients in Eastern India Rate of pain crisis in the HU therapy groups (0.5/Y) was significantly reduced than the control groups (4.8/Y) (p 0.008). Following HU therapy of 2 Years, 95% (19/20) patients become transfusion independent Low dose HU therapy with no significant toxicity seems to be a useful treatment for SCA patients in Eastern India
Jain et al. [32] A double blind randomized controlled trial among SCA children in Central India Event rates per patient per year for VOC, blood transfusions and hospitalization in HU treated group reduced by 95.0, 94.6 and 93.1%, respectively. Also, Hb and Hb F levels were significantly higher in HU treated group than the placebo Significant haematological and clinical benefits of fixed low-dose HU (10 mg/kg/day) therapy were observed among Indian SCA patients
Lakhkar et al. [33] A prospective observational study involving SCA patients from Vidarbha, India 61% of children including SCA children with headache (8%) received 5–10 transfusions/year Transfusion requirement was high in this particular cohort of SCA children
Jain et al. [34] A retrospective analysis involving SCA children from central India Majority (62.0%) of the transfusions were required for SCA children below 5 years of age. Transfusions were mostly given when Hb level drops below 5 g/dl Clinical picture of the SCA children from central India is severe demanding frequent medical attention
Jain et al. [35] A prospective longitudinal study involving SCA children from Nagpur, India Rates of VOC, blood transfusions, sequestration crises, stroke, ACS and rate of hospitalizations were significantly reduced (p < 0.001) after 2 years with HU treatment Low fixed dose HU (10 mg/kg/day) could significantly improve the haematological profile with significant clinical benefits
Mehta et al. [36] A descriptive study of transfusion practice for patients with SCD at a blood bank in southern Gujarat, India During the 18-week evaluation period 145 transfusion were reported for 96 patients. Ten patients (10.4%) received transfusions even with pre-transfusion Hb level ≥ 8 g/dl Transfusions appeared to be widely used among the patients with SCD at the respective centre
Jain et al. [37] Long term observational follow-up study involving Hb SS patients from central India After the HU therapy mean number of VOC, ACS, hospitalization and severe anaemia were reduced in Hb SS patients Long-term low fixed dose HU therapy is efficient in reducing adverse clinical events related to SCD
Oberoi et al. [38] A retrospective study of Hb S-D Punjab patients from Chandigarh, India Only 5 out of 10 patients were on HU therapy. 8 out of 10 patients received transfusion including 1 transfusion dependent patient Encouraging response were noted for HU therapy in Hb S-D Punjab patients
Colah et al. [39] A review of SCD in India Low-fixed dose HU therapy reduced acute clinical events among patients with SCD. Transfusion demand was variable among different sickle phenotypes and communities Low-fixed dose HU therapy is beneficial in ameliorating the severity of Indian SCD
Patel et al. [40] A prospective cohort study involving Hb S-D Punjab patients from Odisha, India HU therapy significantly reduced the VOC and rate of transfusions (p < 0.0001; 0.0008 respectively) among Hb S-D Punjab patients Low-fixed dose HU therapy is effective in reducing VOC and transfusion requirement among Indian Hb S-D Punjab patients
Nimgaonkar et al. [41] A descriptive study of quality of care for patients with SCD from tribal community in Tamil Nadu, India Median annual cost of hospital visits and HU would account for about 18% of the average income of a tribal family. HU was given freely for all the participating patients Financial support is required for patients with SCD from low-income communities in order to implement a sustainable comprehensive care system
Dehury et al. [42] A prospective open label observational study involving Hb S-β+ thalassaemia patients from Odisha, India After the HU therapy, number of blood transfusion per year, VOC & hospitalization reduced significantly (p < 0.0001) Low fixed dose HU therapy is effective in improving the clinical profile of Hb S-β+ thalassaemia patients
Italia et al. [43] A descriptive study involving patients with SCD from central and western India 35% and 39.1% of Hb SS and Hb S-β thalassaemia patients who were classified having severe clinical course had 1–5 times transfusions per year Transfusion requirement appeared to be higher in the more severe SCD than the milder version
Italia et al. [44] A descriptive study evaluating the feasibility of establishing a new-born screening and follow-up programme for SCD in tribal regions of Gujarat, India 3 out of 32 SCD babies received transfusion for severe anaemia (Hb < 6 g/dl). Only 32 out of 46 SCD babies were responding for follow-up Even with multiple attempts to engage with a follow-up programme a proportion of affected SCD babies do not respond neglecting standard care
Upadhye et al. [45] A prospective cohort study involving Hb SS babies from central India Incidence of blood transfusions was 45.1/100 person years. Babies who experienced several stroke episodes were put on chronic transfusion therapy Severe anaemia (Hb < 5 g/dl) and history of stroke potentially required blood transfusions
Serjeant [46] A review discussing a locally appropriate models of care for Indian SCD Anaemic events are frequent in India. Yet, treatment is often empirical with transfusion without detailed examination The role of transfusion therapy should be defined for Indian patients with SCD
Jain et al. [47] A descriptive study involving SCD children from Maharashtra, India Transfusions were marginally more common in Hb S-β thalassaemia patients than in Hb SS patients. All most all hospitalization due to sickle related clinical event resulted in transfusion. Many patients were receiving HU without any documentation of the clinical course A proper guideline should be developed on transfusion practice and usage of HU for Indian patients with SCD
Jain et al. [48] A prospective cohort comparison study involving SCD children from Nagpur, India 24 out of 833 SCD children were on regular transfusion during observation. Median age of starting HU was 12.5 years Systematic implementation of new-born screening, comprehensive care and HU therapy is necessary for Indian patients with SCD
Yadav et al. [49] A retrospective cohort study involving patients with SCD from Jabalpur, India 36.5% patients did not require any blood transfusion during 14-year follow-up period. 16.3% required ≥ 3 transfusions. Transfusions given only when the Hb level dropped < 6.5 g/dl More than 1/3 of the cohort from Jabalpur were able to survive without receiving transfusion for 14 years
Deshpande et al. [20] A single-centre prospective trial involving patients with SCD from Western India After the HU therapy, significant reductions were noted in number pain episodes, transfusion requirement and hospitalization due to pain crisis among both adults and children with SCD HU is beneficial in reducing the pain crisis among patients with SCD thereby improve the quality of the life
Desai et al. [50] A retrospective study involving pregnant women with SCD from Gujarat, India 52.7% of SCD admissions required transfusions and 8.4% admissions had 3 or more transfusions. Blood transfusions were significantly higher among SCD admissions than non SCD admissions (p < 0.01) There is a high risk of adverse outcomes (including transfusions) in SCD pregnancies than non-SCD pregnancies
Mohanty et al. [51] A hospital based analytical cross-sectional study involving adult Hb SS patients from Cuttack, India Even with HU therapy 23 out of 208 patients were on regular transfusions (≥ 3 units/year) while further 32 patients were on occasional transfusion (< 3 units/year) Even with HU therapy demand for transfusions may still persists among Hb SS patients
Sahoo et al. [52] A hospital-based prospective study involving Hb SS patients from Odisha, India Low-fixed dose HU therapy was associated with significant reduction in sperm count, motility and normal morphology (p < 0.0001) Alterations in sperm parameters could be appeared even with low dose HU therapy
Sethy et al. [53] A prospective single centre study involving adult Hb SS patients from Cuttack, India After 3 years of low fixed dose (10 mg/kg/day) HU therapy; number of VOC per year and rate of blood transfusion became significantly lower (p < 0.001) Low fixed dose HU therapy was useful in reducing the VOC and transfusion need among adult Hb SS Patients
Jain and Mohanty [54] A review of clinical manifestation of SCD in India Transfusion requirement is more in Hb S-β thalassaemia. HU therapy was effective in reducing transfusion requirement, pain crisis and hospitalization Whether same management protocol could be practiced across the whole India is questionable. There is a need to deliver suitable guidelines for management of Indian SCD patients
Jariwala et al. [55] A retrospective study involving patients with SCD from Gujarat, India Mean quantity of transfusion was 8.9 units/patient over 8 years. 11% patients developed allo-antibodies Even with < 10 transfusions allo-immunization occurred in patients with SCD. Prevalence of allo-immunization was higher in SCD than in β-thalassaemia major
Dave et al. [56] A longitudinal descriptive study of patients with SCD from tribal area of Gujarat, India SCD comprehensive care programme increased the coverage of HU from 3.5 to 88%. Rate of transfusion reduced significantly after the enrolment with the programme (27.4 vs 17.8 per 100 patient years) Good quality care can be provided even for the economically deprived remote communities with SCD
Somkuwar et al. [57] A prospective cohort study involving Hb SS children from Maharashtra, India After the HU therapy, rate of acute pain crisis and blood transfusion reduced significantly (p = 0.001) Low-fixed dose HU therapy is safe and effective for Indian Hb SS children
Sinha et al. [58] A descriptive survey which projected the blood and budgetary requirement for haemoglobinopathies in India (2017–2026) Annual requirement of blood for SCD would increase by 0.99 million units/year. Projected requirement of blood in 2026 was 9.24 million units Widespread efficient and effective preventive strategies are urgently required to cope with the sharply increasing demand of blood
Jain et al. [59] A prospective cohort comparison study involving Hb SS patients from Nagpur, India 26 (33%) Hb SS patients received 74 transfusions (mean 2.8 episodes/patient) Pre-transfusion Hb was below 6 g/dl in 67% of patients. Only 4 out of 103 Hb SS patients were treated with HU Usage of HU was surprisingly lower among Hb SS patients from Nagpur cohort
Darshana et al. [21] A descriptive cross-sectional study involving patients with SCD from Sri Lanka 33% (3) of Hb SS patients and 5.9% (3) Hb S-β thalassaemia patients were on regular transfusions (> 8 transfusions/year). 26 (43.3%) patients were on HU therapy Usage of HU was not consistent and the practice of transfusions was very variable among Sri Lankan patients with SCD
Barma et al. [60] A prospective cohort study involving SCD children from Odisha, India HU treatment significantly reduced the requirement of blood transfusion (5.4 U/Y to 2.4 U/Y and VOC (p < 0.001). Transfusion rate increased significantly (p < 0.001) among those who were not on HU (5.21 U/Y to 5.94 U/Y) HU therapy could significantly reduce transfusion requirement and VOC among SCD children. Transfusion requirement under no HU therapy may increase with advancing age