Special report

Harif, Mhamed; Weisdorf, DJ; Novitzky, Nicolás; Szer, Jeff; Mahmal, Lahoucine; Benakli, Malek; Othman, Tarek Ben; Bazuaye, Nosakhare Godwin; McGrath, Eoin; Eldridge, Paul W.; Torjemane, Lamia; Madani, Abdellah; Nacer, Redhouane Ahmed; Belkhedim, Reguia; Rasheed, Walid; Ahmed, Syed Osman; Kodera, Yoshihisa; Aljurf, Mahmoud; Niederwieser, Dietger; Quessar, Asmâa

doi:10.1016/j.hemonc.2019.05.003

Cited by 19 publications

(12 citation statements)

References 9 publications

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“…BMT services are already established in six African countries ( Harif et al, 2020 ), but in only one, Nigeria ( Bazuaye et al, 2014 ), of those with a high prevalence of SCD 1 . To date, 17 patients with SCD have received BMT, and 14 have been cured without serious complications 2 .…”

Section: Introductionmentioning

confidence: 99%

“…On one hand, by inhibiting cell division in the later stages of erythropoiesis, it favours the synthesis of foetal haemoglobin (Hb F): insertion of a Hb F molecule hinders polymerization of deoxy-Hb S, thus decreasing sickling ( Faulkner et al, 2021 ). On the other hand, it decreases the neutrophil count, thus reducing the detrimental effects of the chronic inflammatory state that tends to be permanent in SCD patients ( Harif et al, 2020 ).…”

mentioning

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: Introductionmentioning

confidence: 99%

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confidence: 99%

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

Luzzatto

Makani

2022

Front. Pharmacol.

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“…Despite the challenges outlined above, some countries in Africa have managed to establish local programs for cellular therapies. The first meeting of the African Blood and Marrow Transplant Society (AfBMT) was held in 2018 [37].…”

Section: Current Status Of Cellular Therapies In Low-income and Middl...mentioning

confidence: 99%

Global perspectives on cellular therapy for children with sickle cell disease

John

Namazzi

Chirande

et al. 2022

Current Opinion in Hematology

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Purpose of reviewLow-income and middle-income countries (LMICs), primarily in sub-Saharan Africa (SSA), predominantly experience the burden of sickle cell disease (SCD). High frequency of acute and chronic complications leads to increased utilization of healthcare, which burdens fragile health systems. Mortality for children with limited healthcare access remains alarmingly high. Cellular based therapies such as allogeneic hematopoietic stem cell transplant (HSCT) are increasingly used in resource-rich settings as curative therapy for SCD. Broad access to curative therapies for SCD in SSA would dramatically alter the global impact of the disease. Recent findingsCurrently, application of cellular based therapies in LMICs is limited by cost, personnel, and availability of HSCT-specific technologies and supportive care. Despite the challenges, HSCT for SCD is moving forward in LMICs. Highly anticipated gene modification therapies have recently proven well tolerated and feasible in clinical trials in resource-rich countries, but access remains extremely limited.

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“…In this review we describe the current status of HSCT in Tanzania, which does not have an HSCT centre. Currently in central and southern Africa, HSCT centres are located only in Nigeria and South Africa, 7 with countries such as Ghana exploring the possibility of setting up centres. If patients require an HSCT in Tanzania, they are referred to centres abroad which is funded by either personal finances or insurance coverage.…”

Section: Unmet Needs Of Stem Cell Transplantation In Low To Middle Inmentioning

confidence: 99%

“…In central and southern Africa HSCT is available in only two countries, South Africa and Nigeria, for a population of 1 billion Africans. South Africa has an established transplant programme and performs around 250 autologous transplants and 175 allogeneic transplants per year 7 . Nigeria established an allogeneic HSCT programme in 2011 for SCD and has performed six transplants 7 …”

Section: Unmet Needs Of Stem Cell Transplantation In Low To Middle Inmentioning

confidence: 99%