High frequency of acquired aplastic anemia in Tanzania

Ally, Mwashungi; Magesa, Pius; Luzzatto, Lucio

doi:10.1002/ajh.25388

Cited by 11 publications

(13 citation statements)

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“…In an adult cohort from Tanzania, a 42.0% prevalence of PNH clones in AA patients was found. 31 Interestingly, this study also showed an overall higher AA prevalence of six cases per million per year, compared to two per million per year in Europe and North America. A study among a paediatric cohort from Egypt showed the presence of a PNH clone in 36.0% of patients using the CD59 immunohistochemical staining on bone marrow trephine biopsies.…”

Section: Discussionsupporting

confidence: 49%

Childhood aplastic anaemia with paroxysmal nocturnal haemoglobinuria clones: A retrospective single-centre study in South Africa

et al. 2022

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Background: Paroxysmal nocturnal haemoglobinuria (PNH) clones in children are rare but commonly associated with aplastic anaemia (AA) and myelodysplasia.Objective: This study aimed to determine the prevalence of PNH clones in paediatric patients with idiopathic AA, identify differences in clinical and laboratory features and outcomes, and determine the impact of clone size on clinical presentation.Methods: Patients with confirmed idiopathic AA who were tested for PNH between September 2013 and January 2018 at the Inkosi Albert Luthuli Central Hospital, Durban, KwaZulu-Natal, South Africa, were included. PNH clones were detected in neutrophils and monocytes by flow cytometry using fluorescent aerolysin, CD24, CD66b and CD14.Results: Twenty-nine children with AA were identified and 11 were excluded. Ten patients (10/18, 55.6%) had PNH clones ranging from 0.11% to 24%. Compared to the PNH-negative group, these children were older (median: 10 years vs 4 years, p = 0.02) and had significantly lower total white cell counts (median 1.7 × 109/L vs 3.2 × 109/L; p = 0.04). There was no difference in median absolute neutrophil count or haemoglobin concentration. Four patients in each group received immunosuppressive therapy (IST). At six months, all four patients with PNH clones had responded, compared to one in the PNH-negative group.Conclusion: More than half of children with AA had a PNH clone. The size of the clone did not impact clinical severity; however, IST use may positively impact prognosis. We recommend early initiation of IST in patients with AA to avoid delays associated with human leukocyte antigen typing.

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Section: Discussionsupporting

confidence: 49%

Childhood aplastic anaemia with paroxysmal nocturnal haemoglobinuria clones: A retrospective single-centre study in South Africa

et al. 2022

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“…Since PNH is ultra-rare, it is not surprising that there have been very few cases reported from Africa (Manuel, 1969;Oni et al, 1970;Rizk et al, 2002;Lumori and Muyanja, 2019); in Tanzania we have so far diagnosed four patients (Ally et al, 2019). At the moment, as far as we know, ECU is not available in Africa, or in most Asian countries (including India and China), or in most countries in Latin America, and even in some EU countries.…”

Section: Paroxysmal Nocturnal Haemoglobinuriamentioning

confidence: 96%

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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“…Aplastic anaemia (AA) is another condition that can be treated with HSCT. An observational study conducted at a teaching hospital in Dar es Salaam, reported a high incidence of (AA), with a mortality rate of 48%; 15 85% of patients diagnosed had severe or very severe AA, with 80% being diagnosed under the age of 40. The third haematological condition that is a common indication for HSCT is acute leukaemia.…”

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

confidence: 99%