Sickle cell disease (SCD) is a major health issue in sub-Saharan Africa, affecting up to 3% of births in some areas. Around 80% of the 515,000 babies born with SCD each year are in Africa.

Despite the high numbers, many African countries lack advanced treatments that are common in high-income nations, such as exchange blood transfusion (ExBT) and haematopoietic stem cell transplant (HSCT). Developed countries have newborn screening programs to detect SCD early and enroll children in care programs, but such systems are rare in Africa.

Tanzania has taken steps to address this by establishing one of the world’s largest SCD treatment centers. The country has also developed an advanced therapy program focusing on patient engagement, advocacy, ExBT, HSCT, and gene therapy (GT).

Tanzania ranks fifth globally in terms of SCD burden, with around 11,000 affected births annually. A report in BMJ Global Health states that the country has made significant progress in bridging the treatment gap.

Advanced therapies improve SCD patient outcomes and are part of standard care in wealthy countries. The SCD-ATP in Tanzania integrates patient engagement, clinical preparation, gene therapy research, and partnerships, providing a model for other African nations with high SCD cases.

Since 2021, the program has worked on patient-centered initiatives, including workshops and patient camps, to educate and involve patients and caregivers. More than 150 patients, caregivers, and healthcare workers have attended training sessions on ExBT, HSCT, and GT. The program also created a patient registry, screening 1,500 individuals and identifying 157 for possible advanced therapies.

Clinical readiness for advanced therapies was improved by setting up screening protocols using American Society of Haematology guidelines. These guidelines prioritize patients with severe SCD complications like stroke, chronic leg ulcers, and recurrent priapism. As a result, 22 patients received ExBT, and 127 underwent human leukocyte antigen (HLA) typing to find bone marrow donors, creating a sustainable care system.

Ms. Shani Mgaraganza, a mother of two with sickle cell, believes Bone Marrow Transplant (BMT) offers hope. She explains that BMT involves collecting stem cells from the bone marrow and reintroducing them to the patient or a donor.

“It is my request to the government that sickle cell patients be provided with medication to alleviate the challenges of sickle cell disease for free. The medicines they use to reduce the challenges are hydroxyurea and folic acid. These medicines are expensive compared to the income of Tanzanians. For example, one tablet of hydroxyurea costs 1,000/- and there are children who use more than three tablets a day. Who can afford to spend 5,000/- daily to buy medicine for their child for their entire life?” Ms. Mgaraganza questioned.

She called on the government to provide free medication, similar to how ARVs are given to HIV patients.

Dr. Stella Malangahe, Assistant Director of the Department of Haematology/Oncology at Benjamin Mkapa Hospital (BMH), emphasized the urgent need for BMT, especially for children under 12 years old. She explained that SCD treatment is complex, and each BMT patient must stay in the hospital for 40 to 50 days, sometimes longer.

“The treatment process is quite different from others as patients typically need to stay in the hospital for 40 to 50 days, sometimes up to two months. Age consideration is very vital as younger patients have a higher success rate for the procedure, whereas in adults present more challenges,” she said.

Dr. Malangahe added that the government and BMH are working to raise awareness and educate people about SCD treatment.

SCD is a major public health issue in Africa. Tanzania records 11,000 newborn cases annually, and about 14,700 deaths of children under five years old are linked to the disease.

The country has been working on SCD treatment since the 1980s, with Muhimbili National Hospital running a sickle cell clinic. In 2004, Muhimbili University of Health and Allied Sciences (MUHAS) launched a research program that improved healthcare services, advocacy, and training, enrolling 5,466 patients. By 2016, sickle cell services expanded to other health facilities, strengthening care at different levels.

Many African nations prioritized ensuring access to basic care before introducing curative treatments like HSCT. However, advancements in transplantation, increasing demand, and investment in healthcare facilities have accelerated the need for curative strategies.

Tanzania is expanding transplant services in four referral hospitals and using genomic research to explore gene therapy. Over the past two decades, Tanzania has improved early SCD diagnosis and treatment at all levels. At the same time, the country has led initiatives to introduce advanced therapies.

In 2021, the Sickle Cell Programme (SCP) at MUHAS partnered with local and international organizations to start the SCD-ATP. The program aims to strengthen healthcare capacity by training researchers, healthcare providers, and patient advocates, focusing on advanced therapy interventions.

The program identified gaps in advanced healthcare for SCD patients with severe complications. A report highlights the challenges and achievements of setting up SCD-ATP in a low-resource center, describing progress in advocacy, patient engagement, partnerships, gene therapy research, and clinical readiness.

Through local and global collaborations, MUHAS has developed partnerships to support research and clinical programs. The Tanzanian government is committed to improving SCD care, as outlined in its Non-Communicable Disease (NCD) vision for 2026. The HSCT center at Benjamin Mkapa Hospital in Dodoma is part of efforts to expand curative treatments.

BMH, a public-funded hospital, has partnered with the SCD program to facilitate patient referrals for matched sibling donors. Other collaborations include partnerships with the Jakaya Kikwete Cardiac Institute and Muhimbili National Hospital to support SCD treatment.

At the community level, the program worked with patient organizations to design engagement strategies, organize blood donation drives, and support transfusion services. These efforts contribute to the national blood supply, helping patients in need of regular blood transfusions.

Regionally, MUHAS is part of the SickleInAfrica consortium, a network of eight African countries working to develop SCD patient registries. Additionally, MUHAS collaborates with Uganda’s Joint Clinical Research Center to improve regulatory frameworks for gene therapy trials.

At the global level, MUHAS has partnered with various stakeholders to strengthen SCD research and clinical care. The university, in collaboration with the American Society of Gene and Cell Therapy (ASGCT), launched the first Global Gene Therapy Training Program for African educators.