Approximately 40 000 children with sickle cell anemia require screening with TCD and treating with hydroxyurea for stroke prevention in three states in northern Nigeria

Galadanci, Aisha Amal; Galadanci, Najibah A.; Jibir, Binta W.; Abdullahi, Shehu U.; Idris, Nura; Gambo, Safiya; Abubakar, Shehi; Kabo, Nasiru A; Bello‐Manga, Halima; Haliru, Lawal; Bashir, Ibrahim; Aliyu, Muktar H.; Galadanci, Jamil; DeBaun, Michael R.

doi:10.1002/ajh.25616

Cited by 12 publications

(12 citation statements)

References 8 publications

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“…uptake was 100%. In Nigeria a local industry has been sensitive to the demand arising from a 'rare disease' that is not rare in that country, and has knocked down the price to $0.13 (see (Galadanci et al, 2019)): we can hope that others will follow this example.…”

Section: Sickle Cell Diseasementioning

confidence: 99%

“…Indeed, when this medicine was produced locally by galenic compounding and offered to patients free of charge ( Costa et al, 2021 ), the uptake was 100%. In Nigeria a local industry has been sensitive to the demand arising from a ‘rare disease’ that is not rare in that country, and has knocked down the price to $0.13 (see ( Galadanci et al, 2019 )): we can hope that others will follow this example.…”

Section: Introductionmentioning

confidence: 99%

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Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Luzzatto

Makani

2022

Front. Pharmacol.

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Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment. Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous neglect of RD, by providing incentives for development of “orphan drugs” (OD) for their management. Here we analyse the current situation in Africa with respect to (1) sickle cell disease (SCD), that qualifies as rare in the US and in EU, but is not at all rare in African countries (frequencies up to 1–2%); (2) paroxysmal nocturnal haemoglobinuria (PNH), that is ultra-rare in Africa as everywhere else (estimated <10 per million). SCD can be cured by bone marrow transplantation and recently by gene therapy, but very few African patients have access to these expensive procedures; on the other hand, the disease-ameliorating agent hydroxyurea is not expensive, but still the majority of patients in Africa are not receiving it. For PNH, currently most patients In high income countries are treated with a highly effective OD that costs about $400,000 per year per patient: this is not available in Africa. Thus, the impact of OD legislation has been practically nil in this continent. As members of the medical profession and of the human family, we must aim to remove barriers that are essentially financial: especially since countries with rich economies share a history of having exploited African countries. We call on the Global Fund to supply hydroxyurea for all SCD patients; and we call on companies who produce ODs to donate, for every patient who receives an expensive OD in a high income country, enough of the same drug, at a symbolic price, to treat one patient in Africa.

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Section: Sickle Cell Diseasementioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Luzzatto

Makani

2022

Front. Pharmacol.

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“…Marina Cavazzana 2 Kalpna Gupta 3 Obiageli Nnodu 4,5 Isaac Odame 6 Leon Tshilolo 7 Russell Ware 8,9 Lucio Luzzatto 10,11…”

Section: Julie Makaniunclassified

“…Under such a plan hydroxyurea (HU) should be made available free of charge to all patients with SCD. This call is similar to one addressed recently 2 to the President's Emergency Plan for AIDS Relief (PEPFAR: focused until now on HIV‐AIDS); and since a multi‐center trial has provided evidence that HU is highly beneficial in Africa 3 there have been initiatives to provide HU, but they have been local and limited 4–6 The original rationale whereby the Global Fund decided to target the triad of HIV, TB and malaria was that, because they are transmissible diseases, they could be eliminated; however, we seem to be still some way from elimination. SCD is also transmissible, through inheritance rather than through a micro‐organism: and malaria has been the selective agent responsible for the high frequency of SCD in Africa.…”

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confidence: 97%

Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need

et al. 2022

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Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need Progress in understanding human diseases is continuous: by providing new insights it has been a major factor in developing and optimizing the use of old and new drugs. As a result, the numbers of diseases that can be cured or at least significantly modified are increasing continuously. 1 This progress in quality and in quantity has not been matched by progress in the universality of access to care. Vast inequalities in health and in health services exist and persist (see Closing the gap in a generation: health equity through action on the social determinants of health. Geneva, World Health Organization, 2008; and references therein).In the past, for many countries in Africa, the main limiting factor in medical services was failure of diagnosis; nowadays, many of those countries have made efforts to train medical and other health personnel who, having made a diagnosis, are confronted with the need to prescribe drugs that are simply too expensive in relation to the patients' resources. The Orphan Drug Act of 1983 has been a powerful stimulus to the development of new drugs for rare diseases, but the incentives provided by the Act have not been reflected in price structure: as a result, for a majority of patients these diseases have remained orphans. Indeed, inequalities in access have become both greater and more glaring: however, in some cases, there have been initiatives aiming to counter this injustice. The Global Fund, founded in 2002 with the Bill and Melinda Gates being the first and main donors, spends US$4 billion a year, largely in Africa, with the specific objective to defeat HIV, tuberculosis (TB), and malaria.Here we make a call for three concrete steps that would correct at least some of the inequalities in the area of hematology.1. Adding Sickle Cell Disease (SCD) to the triad of conditions for

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“…Children in this cross-sectional study were eligible participants for a National Institute of Health (NIH)-funded randomized controlled trial; the Primary Prevention of Stroke in Children with SCA in sub-Saharan Africa (SPRING Trial, NCT02560935), from July 2016 to July 2017. Participants were recruited from two states in northern Nigeria (estimated population in 2006: 2,828,861) providing medical care for an estimated 32,000 children with SCA [19].…”

Section: Study Design and Populationmentioning

confidence: 99%

World Health Organization’s Growth Reference Overestimates the Prevalence of Severe Malnutrition in Children with Sickle Cell Anemia in Africa

Ghafuri

Abdullahi

Jibir

et al. 2020

JCM

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Anthropometric indices are widely used to assess the health and nutritional status of children. We tested the hypothesis that the 2007 World Health Organization (WHO) reference for assessment of malnutrition in children with sickle cell anemia (SCA) overestimates the prevalence of severe malnutrition when compared to a previously constructed SCA-specific reference. We applied the WHO and SCA-specific references to children with SCA aged 5–12 years living in northern Nigeria (Primary Prevention of Stroke in Children with SCA in sub-Saharan Africa (SPRING) trial) to determine the difference in prevalence of severe malnutrition defined as body mass index (BMI) Z-score <−3 and whether severe malnutrition was associated with lower mean hemoglobin levels or abnormal transcranial Doppler measurements (>200 cm/s). A total of 799 children were included in the final analysis (median age 8.2 years (interquartile range (IQR) 6.4–10.4)). The application of the WHO reference resulted in lower mean BMI than the SCA-specific reference (−2.3 versus −1.2; p < 0.001, respectively). The use of the WHO reference when compared to the SCA-specific reference population also resulted in a higher prevalence of severe malnutrition (28.6% vs. 6.4%; p < 0.001). The WHO reference significantly overestimates the prevalence of severe malnutrition in children with SCA when compared to an SCA-specific reference. Regardless of the reference population, severe malnutrition was not associated with lower mean hemoglobin levels or abnormal transcranial Doppler (TCD) measurements.

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Approximately 40 000 children with sickle cell anemia require screening with TCD and treating with hydroxyurea for stroke prevention in three states in northern Nigeria

Cited by 12 publications

References 8 publications

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet

Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need

World Health Organization’s Growth Reference Overestimates the Prevalence of Severe Malnutrition in Children with Sickle Cell Anemia in Africa

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