PKDL Treatment Trial a Success 

By Thuku Kariuki and Daniel Furnad

In Sudan, a severe skin disease known as PKDL afflicts a large section of the population. Post-kala-azar dermal leishmaniasis, PKDL, develops after one is treated for visceral leishmaniasis (VL).

The disorder is transmitted by sandflies, mainly in East Africa or South Asia. The condition is usually detected when a rash around the mouth appears. Depending on the severity, it can spread to the arms, upper body, and eventually to the entire body.

Currently, the standard treatment for PKDL is sodium stibogluconate (SSG), an injectable drug that is given for a lengthy 60-90 days. It carries life-threatening toxicity when used for an extended period and requires hospital admission, as it must be administered under close supervision.

Fortunately, according to an international study, a hopeful treatment breakthrough has been achieved. The new medication will cut hospital time for PKDL patients in half. It also cuts the amount of time that patients will have to take drugs, something that poses risks in toxicity.  

"Treatment for PKDL in Sudan is currently only recommended for patients with severe or persistent disease, mainly because SSG is prolonged, toxic, and expensive," explains Professor Ahmed Musa, Senior Investigator for Leishmaniasis from the Institute of Endemic Diseases, University of Khartoum.

He hopes the new regimen will encourage an exponential rise in those seeking help.

"We have now found a safer and better treatment option where patients only need to be admitted to hospital for 14 days and then complete the oral treatment at home. This makes it more patient-friendly, which is important since most people affected by this terrible disease are children."

A consortium of concerned organizations came together to improve current treatment for the disease. DNDi, Drugs for Neglected Diseases initiative, and the Institute of Endemic Diseases at the University of Khartoum led the study.

They received financial support from the United Nations World Health Organization (WHO) and several international NGOs. The Phase II trial consisted of two testing arms, starting in 2018 in Soka, Sudan. 90% of the participants were children aged 12 years old and younger.

The first arm saw patients receive a combination of oral miltefosine and injectable paromomycin (MF+PM) for 42 days. They were only hospitalized for 14 days while receiving the medicine. 98% of these sufferers were completely cured. The second arm only required patients to be hospitalized for seven days, while they received a regimen of miltefosine and injectable liposomal amphotericin B (MF+LAmB) for 28 days. These saw an 80% rate of being cured. Both groups were required to take medicine at home after their hospital stays.

Part of what makes this endeavor so heartening is the concern that is shown for these poor victims, who can't rely on more common drugs for other afflictions. The concentration of the disease in poor areas makes it unlikely that any drug company will strike it rich on these cures. However, DNDi has regularly tackled such problems since its inception in 2003. The non-profit has sought out public and private partners to fight disorders in low-income or otherwise neglected areas.  

Besides PKDL, they have taken on sleeping sickness, leishmaniasis, Chagas disease, river blindness, mycetoma, dengue, pediatric HIV, advanced HIV disease, cryptococcal meningitis, and hepatitis C, altogether delivering 13 effective new treatments.

"For a long time, patients with PKDL in Eastern Africa have been left behind by medical research because the disease is not considered life-threatening. Many have had to endure not only stigma but expensive, lengthy treatments exposing them to toxicity,' declares Dr Fabiana Alves, Director of the Leishmaniasis Cluster at DNDi.

She hopes this puts them a step closer to completely ridding affected communities of PKDL: "This new, shorter, and better treatment will improve the lives of these neglected patients and also help reduce VL transmission on our road to elimination."

The reporting regime for new cases in affected areas needs to be strengthened to defeat the disease. The WHO and its partners are trying to ensure that all PKDL cases are detected, reported, and managed by 2030. Complete eradication of the disease could still be decades away, but thousands of patients can now find safe, affordable help.