Rare Diseases Report 2021

2021 Rare Diseases Report: Cancers



Editor's Note: Looking forward
By Mark S. Lesney, PhD
The Rare Diseases Report: Cancers looks to the future from trial design to treatment for some of the most underserved diseases and patient populations.

NORD: Approaching rare cancers through a diversity lens
By Rebecca Aune and Debbie Drell
NORD advocates for all rare disease patients, no matter their race, ethnicity, religion, color, national origin, age, disability, sexual orientation, gender identity, etc.


Precision medicine: A new approach to AML, other blood cancers
By Caleb Rans, PHARMD
Precision medicine makes groundbreaking strides in treating myeloid malignancies by integrating individual molecular data into patient care.

Coping with a shattered immune system: COVID and beyond
By Christine Kilgore
Questions abound about vaccine timing and the immune response in hematologic malignancies.

Racial disparities in blood cancer treatment
By Roxanne Nelson
An increasing number of studies have documented racial disparities among the study, treatment, and management of hematologic malignancies.

Overcoming obstacles in rare GU cancers
By Will Pass
Investigators are meeting the challenge of dealing with rare genitourinary cancers through collaboration, novel treatment efforts, and clinical trial design.

Mesothelioma trials: Moving toward improved survival
By Andrew D. Bowser
New and emerging therapy developments show promise of extended survival for select patients.

Metastatic uveal melanoma -- new drugs in pipeline, but prognoses still grim
By Randy Dotinga
As many as 50% of patients with this rare form of eye cancer develop tumors elsewhere.

Meeting the unmet need in multiple myeloma
By Walter Alexander
Multiple myeloma remains an incurable plasma cell disorder with near-certain relapse after successful treatment.

Rhabdomyosarcoma: Adaptive therapy borrows from nature
By Neil Osterweil
Researchers are applying evolutionary principles to the treatment of childhood, fusion-positive rhabdomyosarcoma.

Recommended Reading

Delays in cancer referral, diagnosis linked with morbidities
MDedge Hematology and Oncology
NHL: As a second-line treatment in phase 3 trial, tisa-cel disappoints
MDedge Hematology and Oncology
ASH studies look at racial disparities in ALL care, outcomes
MDedge Hematology and Oncology
Pembrolizumab improves event-free survival in early TNBC
MDedge Hematology and Oncology
PD-L1 cutoff for pembrolizumab in mTNBC confirmed
MDedge Hematology and Oncology