Health experts in Kenya aim to reduce the number of children born with sickle cell disease — an inherited blood disorder — by more than 12 times through public education and awareness, as the country premiered a guideline for controlling and managing the disease.
The guideline titled Sickle Cell Disease Control and Management and launched recently by the ministry of Health is expected to ensure people living with the condition receive quality and affordable care.
The government has pledged to provide basic treatment medication required by patients like folic acid, hydroxyurea, vaccines, and analgesics for pain management.
Andrew Mulwa, acting director of preventive and promotive health in the Health Ministry, said the guidelines mark a new dawn in the management of sickle cell, which in the past was erratic and relied on individual assessments of health workers across the country.
Sickle cell disease is an inherited blood disorder caused by the presence of abnormal haemoglobin in the red blood cells of affected individuals. Haemoglobin is a protein found in red blood cells that carry oxygen in your body and gives blood its red colour.
Healthy red blood cells are round and move through small blood vessels to carry oxygen to all parts of the body. The United States Centres for Disease Control and Prevention (CDC) said in someone who has sickle cell disease, red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle.”
Normally, red blood cells deliver oxygen throughout the body, thanks to haemoglobin, bending and adjusting to fit into narrow blood vessels, to deliver oxygenated blood to tissues and organs. Rigid sickle cells with abnormal haemoglobin (haemoglobin-HbS), get stuck in the narrow blood vessels preventing oxygenated blood from reaching tissues and organs, causing anaemia that leaves the patient tired and out of breath and sudden attacks of severe pain called sickle cell crises.
Moreover, without adequate oxygenated blood, tissues and organs such as the spleen, liver and kidneys suffer damage over the years, with potentially life-threatening results.
According to experts, lack of early diagnosis prompted by lack of awareness and proper equipment coupled with the high cost of medication used to manage the condition and /or treat complications arising from it has seen many patients, mostly children, and young adults die prematurely. To remedy the situation, the new guideline puts emphasis on early diagnosis and the measures required to reduce the impact of complications and mortality.
“The rationale for this guideline takes cognisant of the challenge patients with sickle cell disease face and the complexities in the provision of healthcare services thus calling for significant health system changes,” said the 50-page document.
Additionally, experts, most of them haematologists (blood specialists) and paediatricians are calling for aggressive public sensitisation to help reduce the numbers from the currently estimated 6,000 newborns (one in every 150 newborns) to less than 500 instead of the projected 10,000 (one in every 100 newborns) annually by 2050.
“Today (July 29), marks a new dawn for us in the fight against sickle cell. We now have guidelines which we hope will see uniformity and standardisation of care across different disciplines,” said Constance Tenge, professor of paediatrics at Moi University School of Medicine.
It is estimated that out of the 240,000 children who are born with sickle cell in Africa annually, 6,000 are in Kenya.
Fredrick Okinyi, chairman of Sickle Cell Federation of Kenya, pushed for the inclusion of drugs such as hydroxyurea, which is on the list of essential medicines under the National Hospital Insurance Fund (NHIF) cover. Dr Okinyi said the drug currently goes for about Ksh50 ($0.46), an amount that is expensive for some patients who require a daily dosage of three drugs.
New drugs meant to reduce the number of painful crises have been developed. In 2017, the US Food and Drug Administration (FDA) approved a drug known as Endari (also called L-glutamine oral powder), to help reduce complications associated with sickle cell disease. The drug is the first treatment approved for patients with sickle cell disease in almost 20 years and is recommended for patients aged five years upwards.
Two years later, the FDA approved a once-a-month drug called Adakveo® (crizanlizumab-tmca) administered as an infusion through the veins to also reduce the frequency of vaso-occlusive crises in adults and paediatric patients, aged 16 years and older, with sickle cell disease. The drug developer, Norvatis, implemented two clinical trials with crizanlizumab in Ghana and Kenya at the International Cancer Institute (ICI) in 2020.
Scientists have been working on a cure for the disease, with reports indicating that two patients appear to have been cured of sickle cell disease after their own genes were edited with CRISPR-Cas9 technology — a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. The two researchers who invented this technology received the Nobel Prize in Chemistry in 2020.
Wataalamu wa afya nchini Kenya wamekusudia kupunguza idadi ya watoto wanaozaliwa na ugonjwa wa seli za mviringo (Sickle cell disease) kwa zaidi ya mara 12 kupitia elimu ya umma na ufahamu, wakati nchi hiyo ilitangaza miongozo ya kudhibiti na kusimamia ugonjwa huo. "Mwongozo wa ""Udhibiti na Usimamizi wa Ugonjwa wa Sickle Cell"" uliotolewa hivi karibuni na Wizara ya Afya unatarajiwa kuhakikisha watu wanaoishi na hali hiyo wanapata huduma bora na ya bei nafuu." Serikali imeahidi kutoa dawa za msingi za matibabu zinazohitajika na wagonjwa kama vile asidi ya foliki, hydroxyurea, chanjo, na dawa za kupunguza maumivu. Andrew Mulwaya, mkurugenzi wa afya ya kuzuia na kukuza katika Wizara ya Afya, alisema miongozo hiyo inaashiria mapumziko mapya katika usimamizi wa seli ya sickle, ambayo hapo awali ilikuwa isiyo ya kawaida na ilitegemea tathmini za kibinafsi za wafanyikazi wa afya nchini kote. Ugonjwa wa seli za mti wa mti ni ugonjwa wa urithi wa damu unaosababishwa na uwepo wa hemoglobini isiyo ya kawaida katika seli nyekundu za damu za watu walioathiriwa. Hemoglobini ni protini inayopatikana katika chembe nyekundu za damu ambazo hubeba oksijeni katika mwili wako na kutoa rangi nyekundu ya damu. Chembe nyekundu za damu zenye afya ni za mviringo na husafiri kupitia mishipa midogo ya damu ili kubeba oksijeni hadi sehemu zote za mwili. "Kampuni ya CDC inasema kwamba kwa mtu aliye na ugonjwa wa seli za mviringo, seli nyekundu za damu huwa ngumu na zenye kunata na zinaonekana kama chombo cha shamba cha umbo la C kinachoitwa ""sickle"" - kwa kawaida, seli nyekundu za damu hutoa oksijeni katika mwili wote, shukrani kwa hemoglobini, na kugeuka na kurekebisha ili kufanana na mishipa nyembamba ya damu." Seli za mviringo zenye kiwango cha juu cha hemoglobini (HbS) huingia kwenye mishipa midogo ya damu na kuzuia damu yenye oksijeni kufikia tishu na viungo, na kusababisha upungufu wa damu ambao humfanya mgonjwa kuwa amechoka na kukosa pumzi na mshtuko wa ghafla wa maumivu makali. Kwa kuongezea, bila damu yenye oksijeni ya kutosha, tishu na viungo kama vile utando, ini na figo hupata uharibifu kwa miaka mingi, na matokeo yanaweza kuhatarisha maisha. Kulingana na wataalamu, ukosefu wa utambuzi wa mapema unaosababishwa na ukosefu wa ufahamu na vifaa sahihi pamoja na gharama kubwa ya dawa zinazotumiwa kusimamia hali hiyo na au kutibu matatizo yanayotokana nayo imeona wagonjwa wengi, hasa watoto na vijana, kufa kabla ya wakati. Ili kurekebisha hali hiyo, miongozo hiyo mpya inasisitiza utambuzi wa mapema na hatua zinazohitajika kupunguza athari za matatizo na vifo. "Ukweli wa mwongozo huu unafahamu changamoto ya wagonjwa wa ugonjwa wa seli ya sickle na utata katika utoaji wa huduma za afya na hivyo inahitaji mabadiliko makubwa ya mfumo wa afya, ""alisema hati ya kurasa 50. Wataalamu wa magonjwa ya damu na watoto wanadai kuwa idadi ya watoto wanaozaliwa kila mwaka itapungua kutoka kwa takriban 6,000 (mmoja kati ya watoto 150) hadi chini ya 500 (mmoja kati ya watoto 10,000) ifikapo mwaka 2050. "Siku ya leo (Julayi 29) inaashiria mapambazuko mapya kwa ajili yetu katika mapambano dhidi ya seli ya sickle. """ """Sasa tuna miongozo ambayo tunatumaini itaona usawa na usawa wa huduma katika taaluma tofauti,"" alisema Constance Tenge, profesa wa pediatrics katika Chuo Kikuu cha Moi School of Medicine." Inakadiriwa kuwa kati ya watoto 240,000 wanaozaliwa na ugonjwa wa seli za mviringo kila mwaka, 6,000 ni wa Kenya. Fredrick Okinyi, mwenyekiti wa Shirikisho la Sickle Cell la Kenya, alisisitiza kuingizwa kwa dawa kama vile hydroxyurea, ambayo iko kwenye orodha ya dawa muhimu chini ya chanjo ya Mfuko wa Bima ya Hospitali ya Kitaifa (NHIF). Daktari Okinyi alisema dawa hiyo kwa sasa inagharimu karibu Ksh50 (Dola 0.46), kiasi ambacho ni ghali kwa baadhi ya wagonjwa ambao wanahitaji kipimo cha kila siku cha dawa tatu. Dawa mpya zinazokusudiwa kupunguza idadi ya matatizo yenye kuumiza zimetengenezwa. Mwaka 2017, Shirika la Chakula na Dawa la Marekani (FDA) lilikubali dawa inayoitwa Endari (iliyoitwa pia L-glutamine oral powder) kusaidia kupunguza matatizo yanayohusiana na ugonjwa wa seli za sickle. Dawa hiyo ni ya kwanza kuidhinishwa kwa wagonjwa wa ugonjwa wa seli za sickle kwa karibu miaka 20 na inashauriwa kwa wagonjwa wenye umri wa miaka mitano au zaidi. Mwaka jana, FDA iliidhinisha dawa ya Advil (Cryzalin, TCM) iliyotolewa mara moja kwa mwezi kupitia mishipa ya damu ili kupunguza mzunguko wa matatizo ya vasoconstriction kwa watu wazima na watoto wenye umri wa miaka 16 na zaidi na ugonjwa wa seli ya sickle. Kampuni ya Norvatis, ambayo ilitengeneza dawa hiyo, ilifanya majaribio mawili ya kliniki ya crizoprazole nchini Ghana na Kenya katika Taasisi ya Kimataifa ya Saratani (ICI) mwaka 2020. Wataalamu wa sayansi wamekuwa wakijaribu kupata tiba ya ugonjwa huo, na ripoti zinaonyesha kuwa wagonjwa wawili wanaonekana kuwa wamepona kutokana na ugonjwa wa seli za mviringo baada ya jeni zao kuhaririwa kwa teknolojia ya CRISPR-Cas9, teknolojia ya kipekee ambayo inawawezesha wataalamu wa jeni na watafiti wa matibabu kuhariri sehemu za genome kwa kuondoa, kuongeza au kubadilisha sehemu za mfululizo wa DNA. Watafiti wawili waliobuni teknolojia hiyo walipata tuzo ya Nobel ya kemia mwaka 2020.