What do PNH patients & PNH doctors want from research? | Aplastic Anemia and MDS International Foundation

What do PNH patients & PNH doctors want from research?

AA&MDSIF recently completed a series of interviews funded by the Geczik Foundation. We asked the opinions of eight patients, and four PNH physician-researchers their thoughts concerning the most important needs for PNH researchers to address.

Patients and physicians we spoke to shared many general concerns related to quality of life, appropriate management and future standards of care for patients diagnosed with PNH. Patients voiced their concern that doctors lacked a fine level understanding about daily quality of life issues and symptom fluctuations they experienced. They also worried about topics like alternatives to the current treatment options, in case long term use of currently available medications became less effective over time or too many unfavorable side-effects developed.

In addition, the patients we interviewed emphasized the need for education of the broader health care community about PNH. This was not only to quicken a PNH diagnosis for other patients, but to alleviate confusion and improper treatment by local non-specialists such as emergency room personnel or others.

The physicians interviewed were more interested having further research on the etiology or cause of PNH, to help them discover different treatment modalities. They were equally concerned about long term treatment effects and interested in having more information and options available to prolong survival for patients. Both patients and physicians were interested in next generation of treatment options.

The next step is for AA&MDSIF to use these findings to draft requests for proposals that will invite and support investigators to address the issues identified and develop solutions. We think 2015 is going to be an exciting year for PNH research!